ATE483817T1 - Adeno-assoziiertes virus mit invertierten terminalen 'repeats' als promoter - Google Patents

Adeno-assoziiertes virus mit invertierten terminalen 'repeats' als promoter

Info

Publication number
ATE483817T1
ATE483817T1 AT01107900T AT01107900T ATE483817T1 AT E483817 T1 ATE483817 T1 AT E483817T1 AT 01107900 T AT01107900 T AT 01107900T AT 01107900 T AT01107900 T AT 01107900T AT E483817 T1 ATE483817 T1 AT E483817T1
Authority
AT
Austria
Prior art keywords
vector
gene
promoter
associated virus
adeno
Prior art date
Application number
AT01107900T
Other languages
English (en)
Inventor
Barrier J Carter
Terrence Flotte
Sandra Afione
Rikki Solow
Original Assignee
Us Gov Health & Human Serv
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Us Gov Health & Human Serv filed Critical Us Gov Health & Human Serv
Application granted granted Critical
Publication of ATE483817T1 publication Critical patent/ATE483817T1/de

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Pulmonology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
AT01107900T 1992-06-02 1993-06-02 Adeno-assoziiertes virus mit invertierten terminalen 'repeats' als promoter ATE483817T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US07/891,962 US5587308A (en) 1992-06-02 1992-06-02 Modified adeno-associated virus vector capable of expression from a novel promoter

Publications (1)

Publication Number Publication Date
ATE483817T1 true ATE483817T1 (de) 2010-10-15

Family

ID=25399130

Family Applications (2)

Application Number Title Priority Date Filing Date
AT93916425T ATE209254T1 (de) 1992-06-02 1993-06-02 Adeno assozierter virus mit umgekehrten terminal repeat sequenzen als promotor fur den in vivo transfer eines funktionellen cftr gens
AT01107900T ATE483817T1 (de) 1992-06-02 1993-06-02 Adeno-assoziiertes virus mit invertierten terminalen 'repeats' als promoter

Family Applications Before (1)

Application Number Title Priority Date Filing Date
AT93916425T ATE209254T1 (de) 1992-06-02 1993-06-02 Adeno assozierter virus mit umgekehrten terminal repeat sequenzen als promotor fur den in vivo transfer eines funktionellen cftr gens

Country Status (10)

Country Link
US (5) US5587308A (de)
EP (2) EP0644944B1 (de)
AT (2) ATE209254T1 (de)
AU (1) AU673367B2 (de)
CA (1) CA2136441C (de)
DE (2) DE69331194T2 (de)
DK (1) DK0644944T3 (de)
ES (1) ES2168096T3 (de)
PT (1) PT644944E (de)
WO (1) WO1993024641A2 (de)

Families Citing this family (547)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5587308A (en) * 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5869305A (en) * 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
US6652850B1 (en) * 1993-09-13 2003-11-25 Aventis Pharmaceuticals Inc. Adeno-associated viral liposomes and their use in transfecting dendritic cells to stimulate specific immunity
US5834441A (en) * 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
FR2716459B1 (fr) * 1994-02-22 1996-05-10 Univ Paris Curie Système hôte-vecteur utilisable en thérapie génique.
FR2716893B1 (fr) * 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
JPH10501686A (ja) * 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのｄｎａのａａｖ仲介送達
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US6342390B1 (en) * 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US6924128B2 (en) * 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6383814B1 (en) * 1994-12-09 2002-05-07 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) * 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6110456A (en) * 1995-06-07 2000-08-29 Yale University Oral delivery or adeno-associated viral vectors
US5646034A (en) * 1995-06-07 1997-07-08 Mamounas; Michael Increasing rAAV titer
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
JPH11510046A (ja) * 1995-07-21 1999-09-07 ローヌ−プーラン ローラー ファーマシューティカルズ インコーポレイテッド アデノ関連ウイルスリポソーム及び樹状細胞をトランスフェクトして特異性免疫を刺激することにおけるそれらの使用
US6207457B1 (en) * 1995-09-08 2001-03-27 Avigen, Inc. Targeted nucleotide sequence delivery and integration system
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
CA2279669A1 (en) * 1995-12-15 1997-06-16 Enzo Therapeutics, Inc. Property effecting and/or property exhibiting constructs for the expression of non-native nucleic acid processing components for therapeutic and diagnostic uses
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
DE19608753C1 (de) * 1996-03-06 1997-06-26 Medigene Gmbh Transduktionssystem und seine Verwendung
US6245735B1 (en) 1996-07-29 2001-06-12 The Brigham And Women's Hospital, Inc. Methods and products for treating pseudomonas infection
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
WO1998048005A1 (en) 1997-04-24 1998-10-29 University Of Washington Targeted gene modification by parvoviral vectors
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6642051B1 (en) * 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
JP2001520047A (ja) * 1997-10-21 2001-10-30 ターゲテッド ジェネティックス コーポレイション 組換えａａｖベクターと共に使用するための転写活性化された逆方向末端反復（ｉｔｒ）
WO1999032647A1 (en) * 1997-12-23 1999-07-01 Introgene B.V. Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
US6294379B1 (en) 1998-02-25 2001-09-25 The Regents Of The University Of California Efficient AAV vectors
AU3456099A (en) 1998-03-31 1999-10-18 Geron Corporation Methods and compositions for eliciting an immune response to a telomerase antigen
DE69918090T2 (de) 1998-04-24 2005-06-16 University Of Florida, Gainesville Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie
ATE309352T1 (de) 1998-05-06 2005-11-15 Vlaams Interuniv Inst Biotech Inhibitoren der nf-kb aktivierung
US6146874A (en) * 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
DE69939169D1 (de) * 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
AU1128400A (en) 1998-10-22 2000-05-08 Medical College Of Georgia Institute, Inc. Long terminal repeat, enhancer, and insulator sequences for use in recombinant vectors
US7129043B1 (en) * 1998-10-22 2006-10-31 Duke University Methods of screening for risk of proliferative disease and methods for the treatment of proliferative disease
US6468793B1 (en) 1998-10-23 2002-10-22 Florida State University Research Foundation CFTR genes and proteins for cystic fibrosis gene therapy
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
EP1135468B1 (de) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
US6537540B1 (en) * 1999-05-28 2003-03-25 Targeted Genetics Corporation Methods and composition for lowering the level of tumor necrosis factor (TNF) in TNF-associated disorders
EP1194449B1 (de) 1999-06-28 2010-09-22 Oklahoma Medical Research Foundation Inhibitoren des memapsin 2 und ihre verwendung
US6596535B1 (en) * 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
JP2003511082A (ja) * 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法
AU2001250774B2 (en) 2000-02-08 2005-01-27 Sangamo Biosciences, Inc. Cells expressing zinc finger proteins for drug discovery
WO2001066782A1 (en) * 2000-03-03 2001-09-13 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
WO2001071018A2 (en) * 2000-03-22 2001-09-27 Board Of Trustees Of The University Of Arkansas Secreting products from skin by adeno-associated virus (aav) gene transfer
US7271150B2 (en) * 2001-05-14 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Modified growth hormone
WO2002094854A2 (en) 2001-05-21 2002-11-28 The Brigham And Women's Hospital, Inc. P.aeruginosa mucoid exopolysaccharide specific binding peptides
US7119172B2 (en) 2001-05-21 2006-10-10 The Brigham And Women's Hospital, Inc. P. aeruginosa mucoid exopolysaccharide specific binding peptides
WO2002100317A2 (en) 2001-05-25 2002-12-19 The Trustees Of The University Of Pennsylvania Targeted particles and methods of using the same
WO2003000280A2 (en) 2001-06-22 2003-01-03 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Abin-mediated hepatitis protection
CA2461290C (en) 2001-09-24 2014-11-25 Sangamo Biosciences, Inc. Modulation of stem cells using zinc finger proteins
CA2472927A1 (en) 2002-01-10 2003-07-17 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw A novel splice variant of myd88 and uses thereof
US7282222B2 (en) 2002-03-15 2007-10-16 The United States Of America As Represented By The Department Of Veterans Affairs Methods and compositions for directing cells to target organs
US6825263B2 (en) * 2002-04-08 2004-11-30 Dow Corning Corporation Curable coating compositions from emulsions of elastomeric polymers and polyurethane dispersions
US20050222061A1 (en) 2002-04-18 2005-10-06 Schulte Ralf W Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
WO2004042024A2 (en) 2002-11-01 2004-05-21 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF HIF-1 ALPHA
ATE546528T1 (de) 2002-11-13 2012-03-15 Univ Jefferson Zusammensetzungen und verfahren zur diagnose und behandlung von krebs
WO2004060346A2 (en) 2002-12-30 2004-07-22 Angiotech International Ag Drug delivery from rapid gelling polymer composition
JP2007524349A (ja) 2003-01-16 2007-08-30 ザ・トラスティーズ・オブ・ザ・ユニバーシティ・オブ・ペンシルバニア ＩＣＡＭ−１のｓｉＲＮＡ阻害のための組成物及び方法
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP2927318B1 (de) 2003-08-08 2020-05-20 Sangamo Therapeutics, Inc. Verfahren und Zusammensetzungen für gezielte Spaltung und Rekombination
CA2539439C (en) 2003-09-19 2012-10-23 Sangamo Biosciences, Inc. Engineered zinc finger proteins for regulation of gene expression
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
EP1713938A2 (de) 2004-02-09 2006-10-25 Thomas Jefferson University Diagnose und behandlung von krebserkrankungen mit oder nahe mit krebs in zusammenhang stehenden chromosomalen merkmalen lokalisierter microrna
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
EP1733034A4 (de) * 2004-03-15 2010-01-20 Biogen Idec Inc Verfahren und konstrukte zur expression von polypeptidmultimeren in eukaryontischen zellen unter vewendung von alternativem spleissen
AU2005233583B2 (en) 2004-04-08 2011-02-03 Sangamo Therapeutics, Inc. Methods and compositions for modulating cardiac contractility
WO2006036465A2 (en) * 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
EP1828390B1 (de) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimäre vektoren
CA2599004C (en) 2005-02-28 2015-05-26 Sangamo Biosciences, Inc. Anti-angiogenic methods and compositions
US8247499B2 (en) 2005-04-22 2012-08-21 Evonik Stockhausen Gmbh Water-absorbing polymer structure with improved absorption properties
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP1913149A4 (de) 2005-07-26 2009-08-05 Sangamo Biosciences Inc Gezielte integration und expression exogener nukleinsäuresequenzen
US8481505B2 (en) 2005-09-12 2013-07-09 The Ohio State University Research Foundation Compositions and methods for the diagnosis and therapy of BCL2-associated cancers
EP2345417A1 (de) 2005-12-22 2011-07-20 Vib Vzw Mittel und Verfahren zur Interferenz mit Proteinen
AU2006337093B2 (en) 2005-12-22 2013-03-14 Opko Pharmaceuticals, Llc. Compositions and methods for regulating complement system
US8669418B2 (en) 2005-12-22 2014-03-11 Vib Vzw Means and methods for mediating protein interference
AU2007205234B2 (en) 2006-01-05 2012-07-12 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis, prognosis and treatment of lung cancer
EP2484782B1 (de) 2006-01-05 2015-11-18 The Ohio State University Research Foundation Verfahren und Zusammensetzungen auf Mikro-RNA-Basis zur Diagnose und Behandlung von soliden Krebs
WO2007081680A2 (en) 2006-01-05 2007-07-19 The Ohio State University Research Foundation Microrna expression abnormalities in pancreatic endocrine and acinar tumors
EP2522748A1 (de) 2006-03-02 2012-11-14 The Ohio State University Mikro-RNA-Expressionsprofil im Zusammenhang mit Bauchspeicheldrüsenkrebs
ES2446362T3 (es) 2006-03-20 2014-03-07 The Ohio State University Research Foundation Huellas de microARN durante megacariocipoyesis humana
JP5704741B2 (ja) 2006-03-31 2015-04-22 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｅｇ５遺伝子発現の抑制のための組成物および方法
WO2007127919A2 (en) 2006-04-28 2007-11-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the jc virus
US7605251B2 (en) 2006-05-11 2009-10-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
EP2392583A1 (de) 2006-05-19 2011-12-07 Alnylam Europe AG. RNAi-Modulation von AHA und therapeutische Verwendungen davon
US7888498B2 (en) 2006-05-22 2011-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
EP2027262B1 (de) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variante foki-spaltungshälften-domänen
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
JP2009540011A (ja) 2006-06-12 2009-11-19 エクセジェニックス、インク．ディー／ビー／エー オプコ ヘルス、インク． 血管新生のｓｉＲＮＡ阻害のための組成物及び方法
ES2425387T3 (es) 2006-07-13 2013-10-15 The Ohio State University Research Foundation Mir-106a para diagnosticar adenocarcinoma de colon de pronóstico de supervivencia pobre
PL2044199T3 (pl) * 2006-07-25 2013-04-30 Celladon Corp Przedłużony, podawany z prądem krwi, nasierdziowy, wieńcowy wlew wektorów wirusowych na bazie wirusów towarzyszących adenowirusom, zawierający SERCA2a do terapii genowej
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
US20090209478A1 (en) 2006-09-21 2009-08-20 Tomoko Nakayama Compositions and methods for inhibiting expression of the hamp gene
US8841436B2 (en) 2007-03-15 2014-09-23 University Hospitals Cleveland Medical Center Screening, diagnosing, treating and prognosis of pathophysiologic status by RNA regulation
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
WO2008116347A1 (en) 2007-03-26 2008-10-02 General Regeneratives Limited Methods for promoting protection and regeneration of bone marrow using cxcl9 and anti-cxcl9 antibodies
JP5350360B2 (ja) 2007-03-29 2013-11-27 アルナイラム ファーマシューティカルズ， インコーポレイテッド エボラ由来の遺伝子の発現を阻害するための組成物および方法
EP2319926B1 (de) 2007-07-05 2016-08-31 Arrowhead Research Corporation DSRNA zur Behandlung viraler Infektionen
AU2008275649B2 (en) 2007-07-12 2013-09-05 Sangamo Therapeutics, Inc. Methods and compositions for inactivating alpha 1,6 fucosyltransferase (FUT 8) gene expression
WO2009012263A2 (en) 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
EP2173911B1 (de) 2007-07-31 2014-06-18 The Ohio State University Research Foundation Verfahren zur methylierungsreversion mittels abzielen auf dnmt3a und dnmt3b
EP2653561B1 (de) 2007-08-03 2016-03-02 The Ohio State University Research Foundation Ultrakonservierte Bereiche zur Codierung von ncRNA
EP3028708A1 (de) 2007-08-22 2016-06-08 The Ohio State University Research Foundation Verfahren und zusammensetzungen zur induzierung von epha7-deregulierung und erk-phosphorlylierung bei humaner akuter leukämie
EP3138926A3 (de) 2007-09-06 2017-04-05 The Ohio State University Research Foundation Mirna-signaturen in menschlichem ovarialkarzinom
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2212440A4 (de) 2007-10-11 2011-04-06 Univ Ohio State Res Found Verfahren und zusammensetzungen zur diagnose und behandlung von adenokarzinomen der speiseröhre
CA2703263C (en) 2007-11-01 2014-03-18 Perseid Therapeutics Llc Immunosuppressive polypeptides and nucleic acids
WO2009076400A2 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
EP2244740A1 (de) 2008-02-19 2010-11-03 Celladon Corporation Zusammensetzungen zur verbesserten aufnahme von virusvektoren in das myokard
CN105267233B (zh) 2008-03-05 2019-07-26 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
JP5763530B2 (ja) 2008-06-10 2015-08-12 サンガモ バイオサイエンシーズ， インコーポレイテッド Ｂａｘ−およびＢａｋ−欠損細胞株の生成のための方法および組成物
JP5908725B2 (ja) 2008-08-22 2016-04-26 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的一本鎖開裂および標的組込みのための方法、並びに組成物
EP2690175B1 (de) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur kombinierten Hemmung der Expression eines mutierenden EGFR-Gens und IL-6
US8546554B2 (en) 2008-09-25 2013-10-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
EP2937418B1 (de) 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur Hemmung der Expression des Transthyretin
EP3269379A1 (de) 2008-11-30 2018-01-17 Immusant, Inc. Zusammensetzungen und verfahren zur behandlung von zöliakie
JP5832293B2 (ja) 2008-12-04 2015-12-16 オプコ ファーマシューティカルズ、エルエルシー 血管新生促進ｖｅｇｆイソ型を選択的に抑制する組成物および方法
EP2373382B1 (de) 2008-12-10 2017-02-15 Alnylam Pharmaceuticals, Inc. Auf gnaq zielende dsrna-zusammensetzungen und verfahren zur unterdrückung der expression
WO2010090744A1 (en) 2009-02-04 2010-08-12 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathies
EP2396343B1 (de) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modifizierte virusvektoren sowie verfahren zu ihrer herstellung und verwendung
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
WO2010105311A1 (en) 2009-03-20 2010-09-23 Angioblast Systems, Inc. Production of reprogrammed pluripotent cells
AU2010235161B2 (en) 2009-04-09 2015-01-22 Sangamo Therapeutics, Inc. Targeted integration into stem cells
SI3988660T1 (sl) 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
EP2451475A2 (de) 2009-07-06 2012-05-16 Novartis AG Selbstreplizierende rna-moleküle und ihre verwendung
EP4183412B1 (de) 2009-07-15 2024-08-14 GlaxoSmithKline Biologicals SA Rsv-f-proteinzusammensetzungen und verfahren zu ihrer herstellung
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
BR112012009801A8 (pt) 2009-10-30 2016-08-30 Cns Therapeutics Inc Polipeptídeo neurturina e variante de neurturina
EP2496607B1 (de) 2009-11-02 2017-09-27 The Regents of The University of California Gewölbte komplexe zur zytokinfreisetzung
US8551781B2 (en) 2009-11-19 2013-10-08 The Regents Of The University Of California Vault complexes for facilitating biomolecule delivery
US8227444B2 (en) 2009-12-04 2012-07-24 Opko Ophthalmics, Llc Compositions and methods for inhibition of VEGF
JP6137596B2 (ja) 2010-02-08 2017-05-31 サンガモ セラピューティクス， インコーポレイテッド 遺伝子操作された切断ハーフドメイン
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US20130281510A1 (en) 2010-03-29 2013-10-24 Kumamoto University siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
EP3578657B1 (de) 2010-04-06 2024-03-20 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der cd274/pd-l1-genexpression
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
EP3456827A3 (de) 2010-06-02 2019-05-08 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung von leberfibrose
JP2013539453A (ja) 2010-07-09 2013-10-24 エクセリクシス， インク． 癌の治療のためのキナーゼ阻害物質の組み合わせ
US9057057B2 (en) 2010-07-27 2015-06-16 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
EP3118316A1 (de) 2010-09-02 2017-01-18 Université de Mons Zur behandlung von fazioskapulohumeraler muskeldystrophie nützliche wirkstoffe
EP3511420B1 (de) 2010-09-27 2021-04-07 Sangamo Therapeutics, Inc. Zusammensetzungen zur hemmung des eindringens von viren in zellen
KR102266691B1 (ko) 2010-10-11 2021-06-23 노파르티스 아게 항원 전달 플랫폼
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
ES3018416T3 (es) 2011-01-26 2025-05-16 Glaxosmithkline Biologicals Sa Régimen de inmunización del VRS
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
KR102104401B1 (ko) 2011-03-29 2020-04-27 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
RS56748B1 (sr) 2011-05-13 2018-03-30 Glaxosmithkline Biologicals Sa Pre-fuzioni rsv f antigeni
EP2714906A1 (de) 2011-06-01 2014-04-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur einstellung der expression von mitochondrialem genom durch microrna
EP2723756B1 (de) 2011-06-21 2020-03-11 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der expression von apolipoprotein-c-iii (apoc3)-genen
EP2723351B1 (de) 2011-06-21 2018-02-14 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der expression von protein-c (proc)-genen
CA2839896A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
US20140235693A1 (en) 2011-06-23 2014-08-21 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
CA2841710C (en) 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
EP2758529B1 (de) 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur regulierung der transgenexpression
BR112014008694A2 (pt) 2011-10-11 2017-06-20 Novartis Ag moléculas de ácido nucleico policistrônico recombinante
US20140348863A1 (en) 2011-10-12 2014-11-27 Alessia Bianchi Cmv antigens and uses thereof
CA2852066A1 (en) 2011-10-14 2013-04-18 The Ohio State University Methods and materials related to ovarian cancer
EP2771482A1 (de) 2011-10-27 2014-09-03 Institut National de la Sante et de la Recherche Medicale (INSERM) Verfahren zur behandlung und diagnose von arteriosklerose
US9375491B2 (en) 2011-10-28 2016-06-28 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
EP2814514B1 (de) 2012-02-16 2017-09-13 Atyr Pharma, Inc. Histidyl-trna-synthetasen zur behandlung von autoimmun- und entzündungserkrankungen
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
WO2013152230A1 (en) 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9193971B2 (en) 2012-04-10 2015-11-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods for the treatment of nonalcoholic steatohepatitis
WO2013153139A1 (en) 2012-04-11 2013-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment and diagnosis of acute leukemia
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
US20150150821A1 (en) 2012-07-09 2015-06-04 The Regents Of The University Of California Vault Immunotherapy
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP2890780B8 (de) 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung eines genetischen leidens
WO2014059173A2 (en) 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
EP3789405A1 (de) 2012-10-12 2021-03-10 The General Hospital Corporation Transkriptionsaktivator-like-effektor-lysinspezifische demethylase-1 (lsd1)-fusionsproteine
BR112015013105B1 (pt) 2012-12-05 2022-02-08 Alnylam Pharmaceuticals, Inc Agente de rnai de fita dupla capaz de inibir a expressão de pcsk9, seus usos, composição farmacêutica e método de inibição da expressão de pcsk9 em uma célula in vitro
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
EP2931899A1 (de) 2012-12-12 2015-10-21 The Broad Institute, Inc. Funktionale genomik unter verwendung von crispr-cas systemen, zusammensetzungen, verfahren, knockout-bibliotheken und anwendungen davon
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
EP3064585B1 (de) 2012-12-12 2020-02-05 The Broad Institute, Inc. Technische bearbeitung und optimierung von verbesserten systemen, verfahren und enzymzusammensetzungen zur sequenzenmanipulation
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP2954042B1 (de) 2013-02-07 2017-12-06 The General Hospital Corporation Tale-transkriptionsaktivatoren
BR112015023108A8 (pt) 2013-03-13 2018-01-23 Geneweave Biosciences Inc sistema de acondicionamento de célula bacteriana, método para acondicionamento de uma molécula de ácido nucléico repórter, composição, método para detectar uma presença ou uma ausência de uma célula bacteriana em uma amostra, vetor, método para detectar um transcrito alvo em uma célula, método para detectar a presença de uma célula bacteriana em uma amostra e kit.
PT2970974T (pt) 2013-03-14 2017-11-29 Alnylam Pharmaceuticals Inc Composições de irna de componente do complemento c5 e métodos para a sua utilização
EP2978446B1 (de) 2013-03-27 2020-03-04 The General Hospital Corporation Anti-cd33 antikörper zur behandlung von morbus alzheimer
EP2997146A4 (de) 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur behandlung eines genetischen leidens
TWI664187B (zh) 2013-05-22 2019-07-01 阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
AU2014268529C1 (en) 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. TMPRSS6 iRNA compositions and methods of use thereof
EP3071598B1 (de) 2013-05-29 2021-07-07 Vybion, Inc. Einkettige intrakörper zur modifizierung des huntingtin-mutanten-abbaus
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
EP3011033B1 (de) 2013-06-17 2020-02-19 The Broad Institute, Inc. Funktionale genomik unter verwendung von crispr-cas-systemen, zusammensetzungen, verfahren, schirme und anwendungen davon
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
MX385871B (es) 2013-10-02 2025-03-18 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
WO2015051318A1 (en) 2013-10-04 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
EP3058072B1 (de) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Freisetzungsverfahren und zusammensetzungen für nukleasevermitteltes genomengineering
MX391904B (es) 2013-11-11 2025-03-12 Sangamo Biosciences Inc Un represor genetico para usarse en el tratamiento de la enfermedad de huntington.
ES2721936T3 (es) 2013-11-13 2019-08-06 Childrens Medical Center Regulación de la expresión genética mediada por las nucleasas
CA3229275A1 (en) 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
WO2015089419A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089368A2 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
EP3079727A1 (de) 2013-12-12 2016-10-19 INSERM - Institut National de la Santé et de la Recherche Médicale Verfahren zur vorbeugung und behandlung von diabetischer kardiomyopathie mithilfe von mir-424/322
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
PT3102673T (pt) 2014-02-03 2020-05-21 Sangamo Biosciences Inc Métodos e composições para tratamento de beta-talassemia
EP3105331B1 (de) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk)-irna-zusammensetzungen und verfahren zur verwendung davon
EP3929279A1 (de) 2014-03-18 2021-12-29 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur regulierung der zinkfingerproteinexpression
BR112016025849A2 (pt) 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
SG11201609376SA (en) 2014-05-22 2016-12-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
AU2015277369B2 (en) 2014-06-16 2021-08-19 The Johns Hopkins University Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
KR102330593B1 (ko) 2014-07-28 2021-11-26 에스케이이노베이션 주식회사 신규 이소프렌 신타아제 및 이를 이용한 이소프렌의 제조방법
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3180426B1 (de) 2014-08-17 2019-12-25 The Broad Institute, Inc. Gemonbearbeitung mit cas9-nickasen
KR101946180B1 (ko) 2014-08-25 2019-02-08 지니위브 바이오사이언시스, 인코포레이티드 비-복제 형질도입 입자 및 형질도입 입자-기반 리포터 시스템
EA201790551A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
CN113699113A (zh) 2014-09-16 2021-11-26 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
TWI864340B (zh) 2014-10-10 2024-12-01 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
EP3215191B1 (de) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc-polynukleotide zur behandlung von morbus parkinson
TWI769565B (zh) 2014-11-10 2022-07-01 美商阿尼拉製藥公司 Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
EP3985115A1 (de) 2014-12-12 2022-04-20 The Broad Institute, Inc. Geschützte guide-rnas (pgrnas)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3230452B1 (de) 2014-12-12 2025-06-11 The Broad Institute, Inc. Deaktivierte guides für crispr-transkriptionsfaktoren
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
RS60762B1 (sr) * 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
CA2981077A1 (en) 2015-04-03 2016-10-06 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
EP3929293A3 (de) 2015-04-03 2022-03-16 University Of Massachusetts Vollständig stabilisierte asymmetrische sirna
SG10202112057QA (en) 2015-05-12 2021-12-30 Sangamo Therapeutics Inc Nuclease-mediated regulation of gene expression
EP3307316A1 (de) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete irna-zusammensetzungen und verfahren zur verwendung davon
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
WO2017024317A2 (en) 2015-08-06 2017-02-09 Dana-Farber Cancer Institute, Inc. Methods to induce targeted protein degradation through bifunctional molecules
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
IL308717A (en) 2015-12-07 2024-01-01 Genzyme Corp Methods and compositions for treating a serpinc1-associated disorder
US11352631B2 (en) 2015-12-18 2022-06-07 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
AU2016374253B2 (en) 2015-12-18 2021-10-21 Sangamo Therapeutics, Inc. Targeted disruption of the MHC cell receptor
KR20180088919A (ko) 2015-12-24 2018-08-07 옥시레인 유케이 리미티드 인간 알파-n-아세틸갈락토사미니다아제 폴리펩티드
EP3402533B1 (de) 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung von neurologischer erkrankung
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
JP7012650B2 (ja) 2016-02-02 2022-01-28 サンガモ セラピューティクス， インコーポレイテッド Ｄｎａ結合ドメインと切断ドメインとを連結するための組成物
AU2017221441A1 (en) 2016-02-17 2018-10-04 Children's Medical Center Corporation FFA1 (GPR40) as a therapeutic target for neural angiogenesis diseases or disorders
EP3445853A1 (de) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1-komplexe mit reduzierter indelaktivität
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
EP3448987A4 (de) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
EP3448874A4 (de) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3469083A1 (de) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete irna-zusammensetzungen und verfahren zur verwendung davon zur behandlung von paroxysmaler nächtlicher hämoglobinurie (pnh)
CN109790551A (zh) 2016-06-16 2019-05-21 奥斯陆大学医院Hf 改进的基因编辑
AU2017283713B2 (en) 2016-06-17 2021-04-08 Massachusetts Institute Of Technology Type VI CRISPR orthologs and systems
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
SG11201900038QA (en) 2016-07-05 2019-02-27 Univ Johns Hopkins Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter
WO2018013840A1 (en) 2016-07-13 2018-01-18 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
CA3030587A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018039440A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger nucleases
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3831281B1 (de) 2016-08-30 2026-02-25 The Regents of The University of California Vorrichtungen und systeme für biomedizinische zielführung und verabreichung
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
WO2018081775A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
EP4276187A3 (de) 2016-12-08 2024-01-17 Case Western Reserve University Verfahren und zusammensetzungen zur erhöhung der herstellung von funktionalem myelin
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3579858A4 (de) 2017-02-07 2020-12-23 The Regents of The University of California Gentherapie für haploinsuffizienz
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
ES2994451T3 (en) 2017-04-20 2025-01-24 Atyr Pharma Inc Compositions for treating lung inflammation
CA3061614A1 (en) 2017-04-28 2018-11-01 The Regents Of The University Of Colorado, A Body Corporate Methods of treating rheumatoid arthritis using rna-guided genome editing of hla gene
EP3618839A4 (de) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
WO2018208910A1 (en) 2017-05-09 2018-11-15 The Broad Institute Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
EP3621979A1 (de) 2017-05-09 2020-03-18 VIB vzw Mittel und verfahren zur behandlung von bakteriellen infektionen
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3625342B1 (de) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systeme, verfahren und zusammensetzungen zur gezielten nukleinsäureeditierung
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
CN111511906A (zh) 2017-06-23 2020-08-07 因思科瑞普特公司 核酸引导性核酸酶
CN111328290A (zh) 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
JP2020530442A (ja) 2017-07-10 2020-10-22 ジェンザイム・コーポレーション 血友病を有する対象の出血事象を処置するための方法および組成物
AU2018301829B2 (en) 2017-07-13 2024-08-01 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase) gene expression
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3658573A1 (de) 2017-07-28 2020-06-03 President and Fellows of Harvard College Verfahren und zusammensetzungen zur entwicklung von basiseditoren unter verwendung von phagenunterstützter kontinuierlicher entwicklung (pace)
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
EP3676376B1 (de) 2017-08-30 2025-01-15 President and Fellows of Harvard College Hocheffiziente baseneditoren mit gam
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
CA3078971A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11008602B2 (en) 2017-12-20 2021-05-18 Roche Molecular Systems, Inc. Non-replicative transduction particles and transduction particle-based reporter systems
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US12269804B2 (en) 2018-01-17 2025-04-08 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
BR112020014140A2 (pt) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated inibidores de dna-pk
WO2019143678A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
WO2019160383A1 (ko) 2018-02-19 2019-08-22 고려대학교 산학협력단 열충격단백질의 에피토프를 포함하는 백신 및 이의 용도
DK3765616T3 (da) 2018-03-14 2023-08-21 Arbor Biotechnologies Inc Nye enzymer og systemer målrettet crispr dna og rna
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
US12636381B2 (en) 2018-04-30 2026-05-26 The Trustees Of The University Of Pennsylvania In utero CRISPR-mediated therapeutic editing of genes
KR102853088B1 (ko) 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
AU2019282149B2 (en) 2018-06-05 2025-08-21 Life Edit Therapeutics, Inc. RNA-guided nucleases and active fragments and variants thereof and methods of use
EP3820495A4 (de) 2018-07-09 2022-07-20 The Broad Institute Inc. Rna-programmierbare epigenetische rna-modifikatoren und deren verwendungen
WO2020018142A1 (en) 2018-07-16 2020-01-23 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
KR102894715B1 (ko) 2018-08-03 2025-12-03 빔 테라퓨틱스, 인크. 핵산 표적 서열을 변형시키기 위한 다중-이펙터 핵염기 편집기 및 이를 이용하는 방법
CA3106035A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Cas12b enzymes and systems
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
EP4234570A3 (de) 2018-09-18 2023-12-13 Sangamo Therapeutics, Inc. Für programmierten zelltod 1 (pd1) spezifische nukleasen
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
IL326834A (en) 2018-10-02 2026-04-01 Sangamo Therapeutics Inc Artificial zinc finger protein transcription factors repressing mapt expression for the prevention and/or treatment of tauopathies
US12285495B2 (en) 2018-10-15 2025-04-29 Fondazione Telethon Ets Genome editing methods and constructs
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
CN113272428A (zh) 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020102659A1 (en) 2018-11-15 2020-05-22 The Broad Institute, Inc. G-to-t base editors and uses thereof
CN113166760A (zh) 2018-11-23 2021-07-23 赛诺菲 用于抑制angptl8的新型rna组合物和方法
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
CN113631704A (zh) 2018-12-27 2021-11-09 生命编辑制药股份有限公司 可用于基因编辑的多肽及使用方法
CN113227404A (zh) 2018-12-27 2021-08-06 豪夫迈·罗氏有限公司 用于检测鲍氏不动杆菌的非复制型转导颗粒和基于转导颗粒的报告系统
US11572595B2 (en) 2018-12-31 2023-02-07 Roche Molecular Systems, Inc. Non-replicative transduction particles with one or more non-native tail fibers and transduction particle-based reporter systems
CN113423419A (zh) 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
WO2020146250A1 (en) 2019-01-07 2020-07-16 Dana-Farber Cancer Institute, Inc. SMALL MOLECULE DEGRADERS OF FKBP12 VIA RECRUITMENT OF VON HIPPEL-LINDAU E3 UBIQUITIN LIGASE (VHL) E3 UBIQUITIN LIGASE, AND USES IN dTAG SYSTEMS
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
WO2020181178A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through thymine alkylation
WO2020181202A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to t:a base editing through adenine deamination and oxidation
WO2020181195A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through adenine excision
WO2020181193A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through adenosine methylation
WO2020181180A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to c:g base editors and uses thereof
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220202957A1 (en) 2019-04-09 2022-06-30 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression
WO2020210751A1 (en) 2019-04-12 2020-10-15 The Broad Institute, Inc. System for genome editing
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP2022529917A (ja) 2019-04-18 2022-06-27 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ガンの処置及び予後のための方法
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
US20210317192A9 (en) 2019-05-29 2021-10-14 Massachusetts Institute Of Technology Hiv-1 specific immunogen compositions and methods of use
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
US20220315906A1 (en) 2019-08-08 2022-10-06 The Broad Institute, Inc. Base editors with diversified targeting scope
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
CN118813654A (zh) 2019-08-12 2024-10-22 生命编辑制药股份有限公司 Rna指导的核酸酶及其活性片段及变体以及使用方法
WO2021030666A1 (en) 2019-08-15 2021-02-18 The Broad Institute, Inc. Base editing by transglycosylation
CN115176011B (zh) 2019-08-27 2025-11-11 赛诺菲 用于抑制pcsk9的组合物和方法
US20220290152A1 (en) 2019-09-03 2022-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CA3153563A1 (en) 2019-09-09 2021-03-18 Beam Therapeutics Inc. Novel crispr enzymes, methods, systems and uses thereof
US20220325296A1 (en) 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
US12503699B2 (en) 2019-10-04 2025-12-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing UGT1a1 gene expression
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
IL292865A (en) 2019-11-13 2022-07-01 Alnylam Pharmaceuticals Inc Methods and preparations for the treatment of angiotensinogen-related disorder - (agt)
WO2021108717A2 (en) 2019-11-26 2021-06-03 The Broad Institute, Inc Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
EP4085133A1 (de) 2019-12-30 2022-11-09 Lifeedit Therapeutics, Inc. Rna-gesteuerte nukleasen, aktive fragmente und varianten davon und verwendungsverfahren
WO2021136752A1 (en) 2019-12-31 2021-07-08 F. Hoffmann-La Roche Ag Quantitative pcr screening of inducible prophage from bacterial isolates
CA3166153A1 (en) 2020-01-28 2021-08-05 The Broad Institute, Inc. Base editors, compositions, and methods for modifying the mitochondrial genome
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
WO2021158999A1 (en) 2020-02-05 2021-08-12 The Broad Institute, Inc. Gene editing methods for treating spinal muscular atrophy
US20230235309A1 (en) 2020-02-05 2023-07-27 The Broad Institute, Inc. Adenine base editors and uses thereof
PH12022552080A1 (en) 2020-02-10 2024-02-12 Alnylam Pharmaceuticals Inc Compositions and methods for silencing vegf-a expression
BR112022017715A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
IL297121A (en) 2020-04-06 2022-12-01 Alnylam Pharmaceuticals Inc Compositions and methods for silencing myoc expression
AU2021251754A1 (en) 2020-04-07 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing SCN9A expression
JP2023516226A (ja) 2020-04-24 2023-04-18 ライフエディット セラピューティクス，インコーポレイティド Ｒｎａ誘導型ヌクレアーゼとその活性な断片およびバリアント、ならびに利用の方法
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
WO2021224395A1 (en) 2020-05-06 2021-11-11 Cellectis S.A. Methods for targeted insertion of exogenous sequences in cellular genomes
JP7891314B2 (ja) 2020-05-06 2026-07-16 セレクティス ソシエテ アノニム 治療用タンパク質の送達のために細胞を遺伝子改変する方法
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CA3173882A1 (en) 2020-05-11 2021-11-18 Alexandra Briner CRAWLEY Rna-guided nucleic acid binding proteins and active fragments and variants thereof and methods of use
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
JP7890972B2 (ja) 2020-07-15 2026-07-15 ライフ エディット セラピューティクス，インコーポレイティド ウラシル安定化タンパク質並びにその活性断片及びバリアント並びに使用方法
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions
EP4700126A3 (de) * 2020-07-23 2026-06-03 The University of North Carolina at Chapel Hill Optimierte slc13a5- gene und expressionskassetten sowie deren verwendung
WO2022034374A2 (en) 2020-08-11 2022-02-17 University Of Oslo Improved gene editing
EP4501951A3 (de) 2020-08-25 2025-04-30 Kite Pharma, Inc. T-zellen mit verbesserter funktionalität
EP4211235A2 (de) 2020-09-11 2023-07-19 Lifeedit Therapeutics, Inc. Dna-modifizierende enzyme und aktive fragmente sowie varianten davon und verfahren zur verwendung
US20220154223A1 (en) 2020-10-15 2022-05-19 Hoffmann-La Roche Inc. Nucleic acid constructs for simultaneous gene activation
WO2022079083A1 (en) 2020-10-15 2022-04-21 F. Hoffmann-La Roche Ag Nucleic acid constructs for va rna transcription
US20230383294A1 (en) 2020-10-16 2023-11-30 Sanofi Novel rna compositions and methods for inhibiting angptl3
EP4229201A1 (de) 2020-10-16 2023-08-23 Sanofi Rna-zusammensetzungen und verfahren zur hemmung von lipoprotein(a)
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
WO2022155500A1 (en) 2021-01-14 2022-07-21 Senti Biosciences, Inc. Secretable payload regulation
CA3173950A1 (en) 2021-03-22 2022-09-22 Tyson D. BOWEN Dna modifyng enzymes and active fragments and variants thereof and methods of use
KR20230158531A (ko) 2021-03-23 2023-11-20 빔 테라퓨틱스, 인크. 신규 crispr 효소, 방법, 시스템 및 그 용도
EP4323501A1 (de) 2021-04-16 2024-02-21 Beam Therapeutics Inc. Genetische modifikation von hepatozyten
EP4341300A1 (de) 2021-05-21 2024-03-27 Cellectis S.A. Erhöhung der wirksamkeit von t-zell-vermittelter immuntherapie durch modulation von krebsassoziierten fibroblasten in soliden tumoren
JP2024522112A (ja) 2021-05-27 2024-06-11 中国科学院動物研究所 エンジニアリングされたＣａｓ１２ｉヌクレアーゼ、エフェクタータンパク質及びその用途
MX2023014008A (es) 2021-05-27 2023-12-11 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion de anhidrasa carbonica 2.
WO2022261509A1 (en) 2021-06-11 2022-12-15 The Broad Institute, Inc. Improved cytosine to guanine base editors
CA3173953A1 (en) 2021-06-11 2023-12-10 Tyson D. BOWEN Rna polymerase iii promoters and methods of use
IL309296A (en) 2021-06-30 2024-02-01 Alnylam Pharmaceuticals Inc Methods and compositions for the treatment of angiotensinogen-related disorder (AGT-)
US20240325568A1 (en) 2021-07-20 2024-10-03 The Broad Institute, Inc. Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
TW202325311A (zh) 2021-08-31 2023-07-01 美商艾拉倫製藥公司 誘導細胞死亡之DFFA樣效應子B (CIDEB) iRNA組合物及其使用方法
KR20260037664A (ko) 2021-09-08 2026-03-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하기 위한 방법 및 조성물
EP4426828A1 (de) 2021-11-01 2024-09-11 Tome Biosciences, Inc. Einzelkonstruktplattform zur gleichzeitigen abgabe von geneditierungsmaschinen und nukleinsäurefracht
CN114015674B (zh) 2021-11-02 2024-08-30 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
AU2022413670A1 (en) 2021-12-17 2024-07-04 Beam Therapeutics Inc. Crispr enzymes, methzods, systems and uses thereof
US20250057980A1 (en) 2021-12-22 2025-02-20 Tome Biosciences, Inc. Co-Delivery of a Gene Editor Construct and a Donor Template
JP2025504898A (ja) 2022-01-24 2025-02-19 ライフエディット セラピューティクス，インコーポレイティド Ｒｎａ誘導型ヌクレアーゼ及びその活性断片及びバリアント、並びに使用方法
AU2023250649A1 (en) 2022-04-04 2024-11-14 The Regents Of The University Of California Genetic complementation compositions and methods
WO2023196772A1 (en) 2022-04-04 2023-10-12 Beam Therapeutics Inc. Novel rna base editing compositions, systems, methods and uses thereof
WO2023196802A1 (en) 2022-04-04 2023-10-12 The Broad Institute, Inc. Cas9 variants having non-canonical pam specificities and uses thereof
EP4508241A1 (de) 2022-04-13 2025-02-19 F. Hoffmann-La Roche AG Verfahren zur bestimmung von aav-genomen
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
EP4522754A1 (de) 2022-05-12 2025-03-19 Sunvax MRNA Therapeutics Inc. Synthetische selbstverstärkende mrna-moleküle mit sekretionsantigen und immunmodulator
EP4526324A1 (de) 2022-05-18 2025-03-26 The Broad Institute, Inc. Manipulierte virale kapside mit erhöhter stabilität und verfahren zur verwendung davon
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
EP4529614A1 (de) 2022-05-23 2025-04-02 F. Hoffmann-La Roche AG Raman-basiertes verfahren zur differenzierung eines aav-partikelserotyps und eines aav-partikelladestatus
EP4532527A1 (de) 2022-05-27 2025-04-09 The Broad Institute, Inc. Verbesserte editoren für mitochondriale basen und verfahren zur bearbeitung mitochondrialer dna
JP2025519228A (ja) 2022-06-03 2025-06-24 エフ． ホフマン－ラ ロシュ アーゲー 組換えａａｖ粒子を生成するための方法
WO2023240137A1 (en) 2022-06-08 2023-12-14 The Board Institute, Inc. Evolved cas14a1 variants, compositions, and methods of making and using same in genome editing
EP4547826A1 (de) 2022-06-30 2025-05-07 Cellectis S.A. Erhöhung der sicherheit einer t-zellvermittelten immuntherapie
EP4554961A2 (de) 2022-07-14 2025-05-21 The Broad Institute, Inc. Aav-kapside zur zns-weiten genverabreichung durch interaktionen mit dem transferrin-rezeptor
WO2024013239A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
AU2023323547A1 (en) 2022-08-12 2025-03-20 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
AU2023325079A1 (en) 2022-08-16 2025-02-13 President And Fellows Of Harvard College Evolved cytosine deaminases and methods of editing dna using same
WO2024042489A1 (en) 2022-08-25 2024-02-29 LifeEDIT Therapeutics, Inc. Chemical modification of guide rnas with locked nucleic acid for rna guided nuclease-mediated gene editing
JP2025530294A (ja) 2022-09-12 2025-09-11 エフ． ホフマン－ラ ロシュ アーゲー 完全なａａｖ粒子と空のａａｖ粒子を分離するための方法
EP4587560A1 (de) 2022-09-16 2025-07-23 Beam Therapeutics Inc. Grosse serinrekombinasen, systeme und verwendungen davon
EP4611823A1 (de) 2022-11-03 2025-09-10 Cellectis S.A. Erhöhung der wirksamkeit und sicherheit einer t-zell-vermittelten immuntherapie
US20260166072A1 (en) 2022-11-04 2026-06-18 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
IL320573A (en) 2022-11-04 2025-07-01 Life Edit Therapeutics Inc Developed adenine deaminases and RNA-guided nuclease fusion proteins with internal insertion sites and methods of use
EP4619524A1 (de) 2022-11-18 2025-09-24 Genkardia Inc. Verfahren und zusammensetzungen zur prävention, behandlung oder umkehrung von diastolischer herzdysfunktion
WO2024127369A1 (en) 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
WO2024127370A1 (en) 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target trac gene and methods of use
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024163842A2 (en) 2023-02-03 2024-08-08 The Broad Institute, Inc. Delivering genes to the brain endothelium to treat lysosomal storage disorder-derived neuropathology
EP4658786A2 (de) 2023-02-03 2025-12-10 The Broad Institute Inc. Geneditierungsverfahren, systeme und zusammensetzungen zur behandlung von spinaler muskelatrophie
AU2024216670A1 (en) 2023-02-07 2025-07-31 F. Hoffmann-La Roche Ag Method for the detection of anti-aav particle antibodies
WO2024194280A1 (en) 2023-03-21 2024-09-26 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
EP4695394A1 (de) 2023-04-12 2026-02-18 Life Edit Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von morbus huntington durch editierung des mutanten huntington-gens
IT202300007968A1 (it) 2023-04-21 2024-10-21 Fond Telethon Ets Metodi di editing genomico e costrutti
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
TW202515898A (zh) 2023-06-30 2025-04-16 日商武田藥品工業股份有限公司 Ｈｔｔ抑制物及其用途
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EP4750890A2 (de) 2023-07-27 2026-06-03 Life Edit Therapeutics, Inc. Rna-geführte nukleasen und aktive fragmente und varianten davon und verfahren zur verwendung
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
CN122422501A (zh) 2023-10-18 2026-07-17 生命编辑治疗股份有限公司 Rna引导的核酸酶及其活性片段和变体以及使用方法
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025122725A1 (en) 2023-12-06 2025-06-12 The Broad Institute, Inc. Methods and compositions for base editing of tpp1 in the treatment of batten disease
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
AU2025218748A1 (en) 2024-02-09 2026-08-06 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025186392A1 (en) 2024-03-06 2025-09-12 Stichting Radboud Universitair Medisch Centrum Therapeutics abca4 genome editing for treatment of stargardt disease
WO2025189168A1 (en) 2024-03-08 2025-09-12 SunVax mRNA Therapeutics Inc. Methods for cascade amplifications of therapeutic payloads (catp) & compositions for cancer immunotherapies and gene therapy
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna
TW202617985A (zh) 2024-06-25 2026-05-01 美商生命編輯治療學公司 新穎反轉錄酶及其用途
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
US20260022401A1 (en) 2024-07-22 2026-01-22 Hoffmann-La Roche Inc. Novel AAV rep ORFs and Rep polypeptides
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
WO2026106945A2 (en) 2024-11-12 2026-05-21 The Broad Institute, Inc. Engineered prime editing guide rnas and methods of making and using same
WO2026132012A1 (en) 2024-12-20 2026-06-25 F. Hoffmann-La Roche Ag Aav particle processing using affinity chromatography with gradient elution
WO2026132139A1 (en) 2024-12-20 2026-06-25 F. Hoffmann-La Roche Ag Purification process for viral vectors
WO2026163162A1 (en) 2025-01-31 2026-08-06 Intellia Therapeutics, Inc. Enhanced cas9 variants and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) * 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) * 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter

Also Published As

Publication number Publication date
US6165781A (en) 2000-12-26
US5587308A (en) 1996-12-24
DK0644944T3 (da) 2002-05-13
EP0644944B1 (de) 2001-11-21
DE69334343D1 (de) 2010-11-18
EP0644944A1 (de) 1995-03-29
US5989540A (en) 1999-11-23
HK1014549A1 (en) 1999-09-30
CA2136441C (en) 2007-04-24
EP1164195B1 (de) 2010-10-06
EP1164195A2 (de) 2001-12-19
ES2168096T3 (es) 2002-06-01
AU4598193A (en) 1993-12-30
WO1993024641A2 (en) 1993-12-09
WO1993024641A3 (en) 1994-05-11
EP1164195A3 (de) 2005-11-23
AU673367B2 (en) 1996-11-07
ATE209254T1 (de) 2001-12-15
PT644944E (pt) 2002-05-31
DE69331194D1 (de) 2002-01-03
DE69331194T2 (de) 2002-07-25
US5990279A (en) 1999-11-23
US5866696A (en) 1999-02-02
CA2136441A1 (en) 1993-12-09

Similar Documents

Publication Publication Date Title
DE69334343D1 (de) Adeno-assoziiertes Virus mit invertierten Terminalen 'Repeats' als Promoter
EP1798239A3 (de) G-Protein-gekoppelte Rezeptoren und deren Verwendung
PT728214E (pt) Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
DK567087D0 (da) Dna-sekvens, der koder for et protein med i alt vaesentligt samme biologiske aktivitet som humanprotein c, samt fremgangsmaade til fremstilling af dette protein
DE60035517D1 (de) Gl50 moleküle, sowie verwendungen derselben
ATE88501T1 (de) Rekombinante dns-expressionsvektoren.
ATE270710T1 (de) Selektion und expression von fremdproteinen mittels eines selektions-amplifikations-systems
NO963358D0 (no) Vertsvektor-system til anvendelse i genterapi
DK42887A (da) Fremgangsmaade til fremstilling af et proteinkompleks med human faktor viii:c aktivitet ved rekombinant-teknik
EP1190043A4 (de) Glykoprotein vi und dessen anwendungen
CY1107629T1 (el) Πρωτεïνη crsp (πλουσιες σε κυστεïνη εκκρινομενες πρωτεïνες) μορια νουκλεϊνικου οξεως που τις κωδικοποιουν και χρησεις τους
ATE93272T1 (de) Expression von protein c.
PT988391E (pt) Vectores adenovirais recombinantes compreendendo uma sequencia de splicing
NZ332897A (en) Methods and compositions for modulating production of a T helper type 2 (Th2)-associated cytokine by regulating transcription factor activity in association with a Nuclear Factor of Activated T cells (NF-AT) protein
EA200000202A1 (ru) Рекомбинантная экспрессия c-пептида инсулина
WO1994013812A3 (en) Novel entomopoxvirus genes, proteins and methods of use thereof
DE60031418D1 (de) 15571, ein neues gpcr-ähnliches molekül der secretin-ähnlichen familie und dessen verwendungen
KR950704477A (ko) 형질 전환된 내피 세포(transformed endothelial cells)
CO5241323A1 (es) Linea celular productora de eritropoyetina humana recombinante .
DE59712771D1 (de) Nebenhoden-spezifisches Rezeptorprotein und dessen Verwendung
KR880701285A (ko) Aids 비루스의 f단백질을 함유하는 백신
EP1243656A3 (de) Baculovirus-Transfervektoren und rekombinante Baculoviren für exogene Genexpression
ES2059431T3 (es) Mutantes de la creatinamidinohidrolasa estables.
ATE181115T1 (de) Stabilisierung von messenger-rns in tierischen zellen
SE9403613D0 (sv) Improvement of an expression vector for production of recombinant proteins

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties