ATE423203T1 - Rekombinantes virales vektorsystem - Google Patents

Rekombinantes virales vektorsystem

Info

Publication number
ATE423203T1
ATE423203T1 AT94904816T AT94904816T ATE423203T1 AT E423203 T1 ATE423203 T1 AT E423203T1 AT 94904816 T AT94904816 T AT 94904816T AT 94904816 T AT94904816 T AT 94904816T AT E423203 T1 ATE423203 T1 AT E423203T1
Authority
AT
Austria
Prior art keywords
recombinant viral
viral vector
vector system
recombinant
encapsidation
Prior art date
Application number
AT94904816T
Other languages
English (en)
Inventor
Richard Samulski
Xiao Xiao
Original Assignee
Univ Pittsburgh
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 Univ Pittsburgh filed Critical Univ Pittsburgh
Application granted granted Critical
Publication of ATE423203T1 publication Critical patent/ATE423203T1/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
    • 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
    • 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

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
AT94904816T 1992-12-04 1993-12-03 Rekombinantes virales vektorsystem ATE423203T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US07/989,841 US5478745A (en) 1992-12-04 1992-12-04 Recombinant viral vector system

Publications (1)

Publication Number Publication Date
ATE423203T1 true ATE423203T1 (de) 2009-03-15

Family

ID=25535518

Family Applications (1)

Application Number Title Priority Date Filing Date
AT94904816T ATE423203T1 (de) 1992-12-04 1993-12-03 Rekombinantes virales vektorsystem

Country Status (9)

Country Link
US (1) US5478745A (de)
EP (1) EP0673415B1 (de)
JP (1) JP4117023B2 (de)
KR (1) KR100256890B1 (de)
AT (1) ATE423203T1 (de)
DE (1) DE69334262D1 (de)
IL (1) IL107862A0 (de)
TW (1) TW260708B (de)
WO (1) WO1994013788A1 (de)

Families Citing this family (525)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5736367A (en) * 1992-03-31 1998-04-07 Medimmune, Inc. Vectors and prokaryotes which autocatalytically delete antibiotic resistance
US5869305A (en) * 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
CA2207927A1 (en) * 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
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
AU763063B2 (en) * 1995-06-07 2003-07-10 University Of North Carolina At Chapel Hill, The AAV transduction of myoblasts
US6040183A (en) * 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
ATE452981T1 (de) * 1995-06-07 2010-01-15 Univ North Carolina Die transduktion von myoblasten mittels vektoren aus adenoassoziierten viren
CA2222574A1 (en) * 1995-06-07 1996-12-19 Yale University Oral delivery of adeno-associated viral vectors
US6093570A (en) * 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
EP0840623B1 (de) 1995-07-21 2007-07-18 Brown University Research Foundation Zusammensetzungen zur gentherapie die nukleinsäure-beladenen polymer-mikropartikeln enthalten
US6248720B1 (en) 1996-07-03 2001-06-19 Brown University Research Foundation Method for gene therapy using nucleic acid loaded polymeric microparticles
US6143211A (en) * 1995-07-21 2000-11-07 Brown University Foundation Process for preparing microparticles through phase inversion phenomena
US6207457B1 (en) 1995-09-08 2001-03-27 Avigen, Inc. Targeted nucleotide sequence delivery and integration system
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
DE19608753C1 (de) * 1996-03-06 1997-06-26 Medigene Gmbh Transduktionssystem und seine Verwendung
EP0927044A4 (de) * 1996-04-16 1999-09-08 Immusol Inc Gerichtete viral vektoren
WO1998009524A1 (en) * 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
EP0931158A1 (de) * 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania Indutierbare methode zur herstellung von rekombinanten adeno-assoziierten viren mittels t7 polymerase
US6544523B1 (en) * 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの産生における使用のための、ａａｖスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
EP0856585A1 (de) * 1997-01-29 1998-08-05 Introgene B.V. Ein induzierbares Replications- und Expressionssystem
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999018227A1 (en) * 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
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
CA2671261A1 (en) 1997-11-06 1999-05-20 Novartis Vaccines And Diagnostics S.R.L. Neisserial antigens
EP1047784B2 (de) 1998-01-14 2015-03-18 Novartis Vaccines and Diagnostics S.r.l. Antigene aus neisseria meningitidis
AU3072799A (en) 1998-03-19 1999-10-11 Human Genome Sciences, Inc. Cytokine receptor common gamma chain like
EP2261338A3 (de) 1998-05-01 2012-01-04 Novartis Vaccines and Diagnostics, Inc. Antigene und Zusammensetzungen gegen Neisseria meningitidis
US6436392B1 (en) * 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
EP1849872A1 (de) * 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adenoassoziierte Virusvektoren und ihre Verwendung
EP1930418B1 (de) 1998-09-04 2015-04-08 Genzyme Corporation Verfahren zur Erzeugung helferfreier Präparate mit hohem Titer aus freigesetzten rekombinanten AAV-Vektoren
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
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
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US20040043488A1 (en) * 1999-02-17 2004-03-04 University Of Pittsburgh Of The Commonwealth System Of Higher Education Adeno-associated viral gene-transfer vector system
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
WO2000066741A2 (en) 1999-04-30 2000-11-09 Chiron S.P.A. Conserved neisserial antigens
GB9911683D0 (en) 1999-05-19 1999-07-21 Chiron Spa Antigenic peptides
DK1180159T3 (da) 1999-05-28 2008-11-17 Targeted Genetics Corp Fremgangsmåder og sammensætninger til at sænke niveauet af Tumor-Nekrose-Faktor (TNF) i TNF-associerede lidelser
EP1939300A1 (de) 1999-05-28 2008-07-02 Targeted Genetics Corporation Verfahren und Zusammensetzung zur Senkung des Spiegels des Tumornekrosefaktors (TNF) bei TNF-assoziierten Leiden
US7122335B1 (en) 1999-06-08 2006-10-17 University Of Iowa Research Foundation Compounds and methods to enhance rAAV transduction
JP4969002B2 (ja) 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
GB9916529D0 (en) 1999-07-14 1999-09-15 Chiron Spa Antigenic peptides
EP1916258B1 (de) 1999-08-09 2014-04-23 Targeted Genetics Corporation Erweiterte Expression einer einstämmigen, heterologen Nukleotidsequenz von rekombinanten viralen Vektoren durch Modellierung der Sequenz mit stammübergreifenden Basispaaren
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
AU7895700A (en) 1999-10-15 2001-04-30 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
JP2004508801A (ja) 1999-10-29 2004-03-25 カイロン　ソチエタ　ア　レスポンサビリタ　リミタータ ナイセリア抗原性ペプチド
PT2289545T (pt) 2000-01-17 2016-09-06 Glaxosmithkline Biologicals Sa Vacina de omv suplementada contra meningococos
US6429001B1 (en) 2000-01-26 2002-08-06 Chiron Corporation Recombinant AAV packaging systems
EP2213743A1 (de) 2000-04-12 2010-08-04 Human Genome Sciences, Inc. Albuminfusionsproteine
CA2881568C (en) 2000-10-27 2019-09-24 Novartis Vaccines And Diagnostics, Inc. Nucleic acids and proteins from streptococcus groups a & b
GB0107658D0 (en) 2001-03-27 2001-05-16 Chiron Spa Streptococcus pneumoniae
GB0107661D0 (en) 2001-03-27 2001-05-16 Chiron Spa Staphylococcus aureus
JP2004536579A (ja) 2001-04-13 2004-12-09 ヒューマン　ジノーム　サイエンシーズ，　インコーポレイテッド 血管内皮増殖因子２
US8067156B2 (en) * 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
CA2456806C (en) * 2001-08-08 2011-10-18 Brown University Research Foundation Methods for micronization of hydrophobic drugs
EP1440091A1 (de) * 2001-10-22 2004-07-28 Novartis AG Nogo rezeptor homologe und deren verwendung
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
WO2003049701A2 (en) * 2001-12-10 2003-06-19 Spherics, Inc. Methods and products useful in the formation and isolation of microparticles
RU2331435C2 (ru) 2001-12-12 2008-08-20 Чирон Срл. Иммунизация против chlamydia trachomatis
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
AU2002364586A1 (en) 2001-12-21 2003-07-30 Delta Biotechnology Limited Albumin fusion proteins
EP1495120B1 (de) 2002-04-18 2012-10-10 Acuity Pharmaceuticals, Inc Mitteln und verfahren zur spezifischen modulation von zielgenen in dem auge
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
NZ540779A (en) 2002-11-01 2008-05-30 Univ Pennsylvania Compositions and methods for siRNA inhibition of HIF-1 alpha
CA2504605C (en) 2002-11-13 2016-01-19 Thomas Jefferson University Treatment of chronic lymphocytic leukemia and prostate cancer with microrna mir15
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
JP2006518997A (ja) * 2003-01-21 2006-08-24 ブリストル−マイヤーズ　スクイブ　カンパニー 新規アシルコエンザイムａ：モノアシルグリセロールアシルトランスフェラーゼ−３（ｍｇａｔ３）をコードするポリヌクレオチドおよびその用途
AU2004227358B8 (en) 2003-03-31 2009-12-10 Targeted Genetics Corporation Compounds and methods to enhance rAAV transduction
GB0308198D0 (en) 2003-04-09 2003-05-14 Chiron Srl ADP-ribosylating bacterial toxin
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
EP2345731B1 (de) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adenoassoziierte Virus-Claden, Sequenzen, diese enthaltende Vektoren und ihre Verwendungen
CA2554818A1 (en) 2004-02-09 2005-08-25 Thomas Jefferson University Diagnosis and treatment of cancers with microrna located in or near cancer-associated chromosomal features
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
JP4792390B2 (ja) 2004-03-29 2011-10-12 株式会社ガルファーマ 新規ガレクチン９改変体タンパク質及びその用途
WO2006012625A2 (en) * 2004-07-22 2006-02-02 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Stat3 decoy oligonucleotides and uses therefor
JP5136766B2 (ja) * 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP2009507918A (ja) 2005-09-12 2009-02-26 ジ・オハイオ・ステイト・ユニバーシティ・リサーチ・ファウンデイション Ｂｃｌ２関連癌の診断及び療法のための組成物及び方法
AU2006337093B2 (en) 2005-12-22 2013-03-14 Opko Pharmaceuticals, Llc. Compositions and methods for regulating complement system
EP1969147B1 (de) 2006-01-05 2014-07-30 The Ohio State University Research Foundation mikro-RNA-basierte verfahren zur Diagnose von Magenkrebs
ES2553442T3 (es) 2006-01-05 2015-12-09 The Ohio State University Research Foundation Procedimientos basados en los microARN para el diagnóstico, pronóstico y tratamiento del cáncer de pulmón
US7670840B2 (en) 2006-01-05 2010-03-02 The Ohio State University Research Foundation Micro-RNA expression abnormalities of pancreatic, endocrine and acinar tumors
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
EP2522746B1 (de) 2006-03-02 2014-11-12 The Ohio State University Research Foundation Mikro-RNA-Expressionsprofil im Zusammenhang mit Bauchspeicheldrüsenkrebs
EP2369011A1 (de) 2006-03-20 2011-09-28 The Ohio State University Research Foundation Miko-RNA-Fingerabdrücke während humaner Megakaryozytopoiese
CA2647769C (en) 2006-03-20 2015-05-26 Japan Science And Technology Agency Control of intracellular target molecule by ip3 receptor-binding protein
NZ571568A (en) 2006-03-31 2010-11-26 Alnylam Pharmaceuticals Inc Double-stranded RNA molecule compositions and methods for inhibiting expression of Eg5 gene
WO2007127919A2 (en) 2006-04-28 2007-11-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the jc virus
CN101484588B (zh) 2006-05-11 2013-11-06 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
CN101489566B (zh) 2006-05-19 2012-04-18 阿尔尼拉姆医药品有限公司 Aha基因的RNAi调控及其治疗性应用
WO2007137220A2 (en) 2006-05-22 2007-11-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of ikk-b gene
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
PT2054431E (pt) 2006-06-09 2011-11-03 Novartis Ag Confórmeros de adesinas bacterianas
DK2029746T3 (da) 2006-06-12 2012-10-08 Exegenics Inc D B A Opko Health Inc Sammensætninger og fremgangsmåder til siRNA-hæmning af angiogenese
ES2535877T3 (es) 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
US7572618B2 (en) 2006-06-30 2009-08-11 Bristol-Myers Squibb Company Polynucleotides encoding novel PCSK9 variants
EP2041317A4 (de) 2006-07-13 2009-10-14 Univ Ohio State Res Found Mikro-rna-basierte verfahren und zusammensetzungen zur diagnose und behandlung von erkrankungen in zusammenhang mit darmkrebs
GB2437893A (en) * 2006-07-25 2007-11-07 Celladon Corp Extended antegrade epicardial coronary infusion of adeno-associated viral vectors for gene therapy
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
EP2066687A4 (de) 2006-09-21 2010-12-08 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur hemmung der hamp-genexpression
WO2008115387A2 (en) 2007-03-15 2008-09-25 University Hospitals Of Cleveland Screening, diagnosing, treating and prognosis of pathophysiologic states 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
EP2905336A1 (de) 2007-03-29 2015-08-12 Alnylam Pharmaceuticals Inc. Zusammensetzungen und Verfahren zur Hemmung der Expression eines Gens von Ebola
EP2316943B1 (de) 2007-07-05 2013-06-19 Novartis AG DSRNA zur Behandlung viraler Infektionen
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
CN101809169B (zh) 2007-07-31 2013-07-17 俄亥俄州立大学研究基金会 通过靶向dnmt3a和dnmt3b恢复甲基化的方法
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
EP2775001B1 (de) 2007-09-06 2016-03-09 The Ohio State University Research Foundation MikroRNA-Signaturen in menschlichem Ovarialkarzinom
EP2212440A4 (de) 2007-10-11 2011-04-06 Univ Ohio State Res Found Verfahren und zusammensetzungen zur diagnose und behandlung von adenokarzinomen der speiseröhre
CA2707042A1 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
WO2009105135A1 (en) 2008-02-19 2009-08-27 Celladon Corporation Compositions for enhanced uptake of viral vectors in the myocardium
KR101397407B1 (ko) 2008-03-05 2014-06-19 알닐람 파마슈티칼스 인코포레이티드 Ｅｇ5 및 ＶＥＧＦ 유전자의 발현을 억제하기 위한 조성물 및 방법
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010028054A1 (en) 2008-09-02 2010-03-11 Alnylam Europe Ag. Compositions and methods for inhibiting expression of mutant egfr gene
AU2009296395A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
MX360460B (es) 2008-10-20 2018-11-05 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
AU2009322279A1 (en) 2008-12-04 2011-07-14 Opko Pharmaceuticals, Llc Compositions and methods for selective inhibition of pro-angiogenic VEGF isoforms
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
AU2010223967B2 (en) 2009-03-12 2015-07-30 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
EP2425000B1 (de) 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Zusammensetzungen für das targeting von zellen der konduktiven luftwege, die konstrukte des adenoassoziierten virus enthalten
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
AP2015008874A0 (en) 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
CN102695723A (zh) 2009-10-30 2012-09-26 中枢神经系统治疗学公司 改进的神经营养因子分子
CN105031618A (zh) 2009-11-02 2015-11-11 加利福尼亚大学董事会 用于细胞因子递送的穹窿体复合物
BR112012010755A2 (pt) 2009-11-05 2015-09-22 Proyecto Biomedicina Cima Sl constructo de gene que permite a expressão hepato-específica induzível de um polinucleotídeo de interesse em resposta a um agente indutor, vetor, genoma viral recombinante, vírion, método in vitro para a expressão de um polinucleotídeo de interesse em uma célula de origem hepática, composição farmacêutica e operador-promotor bi-direcional induzível adequado para a expressão hepato-específica induzível de dois polinucleotídeos de interesse por um agente indutor
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
WO2011084694A1 (en) 2009-12-17 2011-07-14 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Stabilized stat3 decoy oligonucleotides and uses therefor
EP2524037B1 (de) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restriktive umgekehrte endverstärkungen für virusvektoren
AU2011235276B2 (en) 2010-03-29 2015-09-03 Alnylam Pharmaceuticals, Inc. SiRNA therapy for transthyretin (TTR) related ocular amyloidosis
EP2555778A4 (de) 2010-04-06 2014-05-21 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur hemmung der cd274/pd-l1-genexpression
US8993532B2 (en) 2010-04-23 2015-03-31 Cold Spring Harbor Laboratory Structurally designed shRNAs
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
EP3444346B1 (de) 2010-04-23 2022-07-27 University of Massachusetts Aav-basierte behandlung von cholesterinerkrankungen
EP2561073B1 (de) 2010-04-23 2016-08-24 University of Massachusetts Auf das zentralnervensystem abzielende vektoren und verfahren zu ihrer verwendung
CA3102008A1 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
PH12013500064A1 (en) 2010-07-09 2013-03-11 Exelixis Inc Combinations of kinase inhibitors for the treatment of cancer
EP2605798A1 (de) 2010-07-12 2013-06-26 Universidad Autònoma de Barcelona Gentherapiezusammensetzung für die behandlung von diabetes
ES2596977T3 (es) 2010-09-02 2017-01-13 Université de Mons Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral
EP2649182A4 (de) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur erhöhung einer erythropoietin (epo)-herstellung
EP2648763A4 (de) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur expressionshemmung der gene klf-1 und bcl11a
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
PH12013501969B1 (en) 2011-03-29 2018-08-31 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of tmprss6 gene
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
EP3318635A1 (de) 2011-04-21 2018-05-09 University of Massachusetts Raav-basierte zusammensetzungen und verfahren zur behandlung von alpha-1-anti-trypsin-mangel
US20140134728A1 (en) 2011-06-01 2014-05-15 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods for adjusting expression of mitochondrial genome by microrna
EP3388068A1 (de) 2011-06-21 2018-10-17 Alnylam Pharmaceuticals, Inc. Zusammensetzung und verfahren zur hemmung der expression von protein-c (proc)-genen
MX390699B (es) 2011-06-21 2025-03-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3).
US20140275211A1 (en) 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
EP2723758B1 (de) 2011-06-21 2018-06-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-3 (angptl3)-irna-zusammensetzungen und verwendungsverfahren dafür
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
EP2739735A2 (de) 2011-08-01 2014-06-11 Alnylam Pharmaceuticals, Inc. Verfahren zur verbesserung der erfolgsquote von hämatopoetischen stammzellentransplantaten
EP2766500A4 (de) 2011-10-14 2015-10-14 Univ Ohio State Verfahren und materialien in verbindung mit eierstockkrebs
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
EP2814514B1 (de) 2012-02-16 2017-09-13 Atyr Pharma, Inc. Histidyl-trna-synthetasen zur behandlung von autoimmun- und entzündungserkrankungen
US20150190532A1 (en) 2012-04-04 2015-07-09 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
JP2015518475A (ja) 2012-04-10 2015-07-02 インサーム（インスティテュ ナシオナル ドゥ ラ サンテ エ ドゥ ラルシェルシェ メディカル）Ｉｎｓｅｒｍ（Ｉｎｓｔｉｔｕｔ Ｎａｔｉｏｎａｌ Ｄｅｌａ Ｓａｎｔｅ Ｅｔｄｅ Ｌａ Ｒｅｃｈｅｒｃｈｅ Ｍｅｄｉｃａｌｅ） 非アルコール性脂肪性肝炎の治療方法
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
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
US20150150821A1 (en) 2012-07-09 2015-06-04 The Regents Of The University Of California Vault Immunotherapy
EP2872183B1 (de) 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-vermittelte gentherapie zur x-chromosomalen netzhautdegeneration
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
EP2900686B1 (de) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Gegen oligodendrozyten gerichtete aav-vektoren
IL292159B1 (en) 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
WO2014120975A1 (en) 2013-02-01 2014-08-07 California Institute Of Technology Antibody-mediated immunocontraception
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
DK2968424T3 (da) 2013-03-13 2020-03-30 Geneweave Biosciences Inc Ikke-replikative transduktionspartikler og transduktionspartikelbaserede reportersystemer
ES2667209T3 (es) 2013-03-14 2018-05-10 Asklepios Biopharmaceutical, Inc. Genes del receptor-1 de VEGF soluble modificado y vectores para terapia génica
IL288931B2 (en) 2013-03-14 2025-05-01 Alnylam Pharmaceuticals Inc Compositions comprising a complementary portion of C5 IRNA and methods of using them
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US9937231B2 (en) 2013-03-27 2018-04-10 The General Hospital Corporation Methods and agents for treating Alzheimer's disease
WO2014168953A1 (en) 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
EA038792B1 (ru) 2013-05-22 2021-10-20 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
KR102234623B1 (ko) 2013-05-22 2021-04-02 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
CN111205369B (zh) 2013-05-29 2024-03-08 维拜昂公司 改变亨廷顿蛋白突变体降解的单链胞内抗体
LT3013855T (lt) 2013-06-24 2021-01-25 Xiao, Weidong Mutavusio viii faktoriaus kompozicijos ir metodai
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
CA2925107A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
LT3052628T (lt) 2013-10-04 2020-09-10 Alnylam Pharmaceuticals, Inc. Kompozicijos ir būdai alas1 geno raiškai nuslopinti
SG10201804960RA (en) 2013-12-12 2018-07-30 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
WO2015086828A1 (en) 2013-12-12 2015-06-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the prevention and treatment of diabetic cardiomyopathy using mir-424/322
CN113057959B (zh) 2014-02-11 2024-07-16 阿尔尼拉姆医药品有限公司 己酮糖激酶（KHK）iRNA组合物及其使用方法
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
CA2942515C (en) 2014-03-18 2025-12-09 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
EP3134431B1 (de) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr-varianten und deren verwendung in zusammensetzungen zur verringerung des cholesterinspiegels
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
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
AU2015264038B2 (en) 2014-05-22 2021-02-11 Alnylam Pharmaceuticals, Inc. Angiotensinogen (AGT) iRNA compositions and methods of use thereof
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
ES2778727T3 (es) 2014-08-25 2020-08-11 Geneweave Biosciences Inc Partículas de transducción no replicativas y sistemas indicadores basados en partículas de transducción
JP7242180B2 (ja) 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
WO2016037931A1 (en) 2014-09-11 2016-03-17 Fundación Para La Investigación Médica Aplicada Nucleic acid constructs and expression vectors for gene therapy of acute porphyrias and other diseases
EP3200830B1 (de) 2014-10-03 2020-09-09 University of Massachusetts Hocheffiziente bibliotheksidentifizierte aav-vektoren
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
TW202503057A (zh) 2014-10-10 2025-01-16 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
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
KR102545316B1 (ko) 2014-11-10 2023-06-22 알닐람 파마슈티칼스 인코포레이티드 Ｂ형 간염 바이러스(ｈｂｖ) ｉｒｎａ 조성물 및 그의 이용 방법
JP2017535552A (ja) 2014-11-17 2017-11-30 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． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
EP4344741A3 (de) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Auf das zentrale nervensystem gerichtete aav-vektoren
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US11033638B2 (en) 2015-01-07 2021-06-15 Universität Autonoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
US10907176B2 (en) 2015-01-14 2021-02-02 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
MX388739B (es) 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
EP3256170B1 (de) 2015-02-13 2020-09-23 University of Massachusetts Zusammensetzungen und verfahren zur transitorischen freisetzung von polypeptiden
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
KR20240119152A (ko) 2015-03-10 2024-08-06 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 재조합 glut1 아데노-관련 바이러스 벡터 작제물 및 glut1 발현을 회복하기 위한 관련된 방법
EP3285788B1 (de) 2015-04-23 2024-12-18 University of Massachusetts Modulation der transgenexpression eines aav-vektors
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
EP3294309A4 (de) 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. Nukleinsäuremoleküle mit abstandshaltern und verfahren zur verwendung davon
HK1256341A1 (zh) 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
US10966414B2 (en) 2015-05-26 2021-04-06 California Institute Of Technology Population control using engineered translocations
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
CN108291216B (zh) 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
CA3001594A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3364997B1 (de) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gentherapie zur behandlung von morbus canavan
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
SG11201802408RA (en) 2015-11-05 2018-05-30 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
KR20180095843A (ko) 2015-12-07 2018-08-28 젠자임 코포레이션 Serpinc1-연관 장애의 치료를 위한 방법 및 조성물
AU2016366560A1 (en) 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JP7061067B2 (ja) 2015-12-14 2022-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クリグラー・ナジャー症候群の処置のための組成物
JP7406783B2 (ja) 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
CA3008264A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
MX2018007330A (es) * 2015-12-15 2019-03-14 Genzyme Corp Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
EP3393501B1 (de) 2015-12-24 2023-06-07 Oxyrane UK Limited Menschliches alpha-n-acetylgalactosaminidase-polypeptid
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
EP3411484B1 (de) 2016-02-05 2023-10-04 Emory University Injektion von einzelsträngigem oder selbstkomplementärem adeno-assoziiertem virus 9 in den liquor
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
CN109475756A (zh) 2016-02-17 2019-03-15 儿童医疗中心有限公司 Ffa1(gpr40)作为神经血管生成疾病或病症的治疗标靶
CA3011943A1 (en) 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
CA3016985C (en) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11773410B2 (en) 2016-04-13 2023-10-03 University Of Massachusetts Repairing compound heterozygous recessive mutations by allele exchange
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
SG11201808812RA (en) 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
EP3449250B1 (de) 2016-04-28 2020-11-04 Indiana University Research & Technology Corporation Verfahren und zusammensetzungen zur auflösung von komponenten einer viruspräparation
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
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)
CN109415730A (zh) 2016-06-13 2019-03-01 北卡罗来纳大学教堂山分校 优化的cln1基因和表达盒以及它们的应用
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JP7493334B2 (ja) 2016-07-05 2024-05-31 ユニバーシティ オブ マサチューセッツ 緑内障における神経保護療法としてのｓＦａｓＬのＡＡＶ２媒介遺伝子送達
WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
BR112019001344A2 (pt) 2016-07-26 2019-04-30 The University Of North Carolina At Chapel Hill tolerância imunológica mediada por vetor no olho
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
JP2020500928A (ja) 2016-11-15 2020-01-16 リジェネクスバイオ インコーポレイテッド Ｍｐｓｉおよびｍｐｓｉｉならびに他の神経障害において神経機能を改善するための方法
WO2018112320A1 (en) 2016-12-16 2018-06-21 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
MX2019008143A (es) 2017-01-07 2020-01-13 Selecta Biosciences Inc Dosificación sistemática de inmunosupresores acoplados a nanoportadores sintéticos.
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
JP2020510648A (ja) 2017-02-20 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を処置するための遺伝子治療
UY37620A (es) 2017-02-28 2018-08-31 Univ Pennsylvania Vacunas contra la gripe mediadas por aav novedosas
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
RU2019130004A (ru) 2017-03-01 2021-04-01 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия при глазных заболеваниях
EP3592853A1 (de) 2017-03-09 2020-01-15 President and Fellows of Harvard College Unterdrückung von schmerzen durch geneditierung
NZ756587A (en) 2017-03-15 2025-12-19 Univ North Carolina Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
CN110709095B (zh) 2017-04-05 2024-05-03 马萨诸塞大学 小基因治疗
CA3060514A1 (en) 2017-04-20 2018-10-25 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
IL316926A (en) 2017-05-11 2025-01-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
JP7235676B2 (ja) 2017-05-17 2023-03-08 ザ ジェネラル ホスピタル コーポレイション 結節性硬化症の遺伝子治療
IL270882B2 (en) 2017-05-24 2024-02-01 Univ Barcelona Autonoma Viral expression vectors containing the coding sequence for FIBROBLAST GROWTH FACTOR 21 (FGF21
EP3630986B1 (de) 2017-05-31 2026-03-25 The Trustees of The University of Pennsylvania Gentherapie zur behandlung von peroxisomalen erkrankungen
JP7766393B2 (ja) 2017-06-14 2025-11-10 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
TW202434265A (zh) 2017-07-10 2024-09-01 美商健贊公司 於患有血友病之個體中治療出血事件之方法及組成物
KR102696307B1 (ko) * 2017-09-08 2024-08-16 제너레이션 바이오 컴퍼니 비-바이러스성 캡시드-비함유 dna 벡터의 지질 나노입자 제형
WO2019060538A1 (en) 2017-09-20 2019-03-28 The Trustees Of Indiana University METHODS FOR LIPOPROTEIN RESOLUTION BY MASS SPECTROMETRY
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
US12441998B2 (en) 2017-10-12 2025-10-14 The Trustees Of Columbia University In The City Of New York SLC2A1 lncRNA as a biologic and related treatments and methods
BR112020007157A2 (pt) 2017-10-13 2020-09-24 Selecta Biosciences, Inc. métodos e composições para a atenuação de respostas de igm antivetor de transferência viral
US12043848B2 (en) 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
SG11202002940QA (en) 2017-11-01 2020-04-29 Alnylam Pharmaceuticals Inc Complement component c3 irna compositions and methods of use thereof
RU2020118342A (ru) 2017-11-07 2021-12-08 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены aga и экспрессионные кластеры и их применение
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
EP3701031A4 (de) 2017-11-28 2021-08-11 Mirimus, Inc. Verfahren zur genetischen vermittelten manipulation von rnai-modellen
EP3727468A4 (de) 2017-12-19 2021-09-22 Akouos, Inc. Aav-vermittelte abgabe von therapeutischen antikörpern an das innenohr
US11008602B2 (en) 2017-12-20 2021-05-18 Roche Molecular Systems, Inc. Non-replicative transduction particles and transduction particle-based reporter systems
EP3738137A1 (de) 2018-01-12 2020-11-18 The Trustees of Indiana University Konstruktion einer elektrostatischen linearen ionenfalle für massenspektrometrie mit ladungsdetektion
CA3088323A1 (en) 2018-01-17 2019-07-25 Adrenas Therapeutics, Inc. Adeno-associated virus gene therapy for 21-hydroxylase deficiency
EP3740580A4 (de) 2018-01-19 2021-10-20 Duke University Genom-engineering mit crispr-cas-systemen bei eukaryonten
EP3759218A4 (de) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für antikörperausweichende virusvektoren
EP3758736B1 (de) 2018-03-02 2024-05-01 The United States of America, as represented by The Secretary, Department of Health and Human Services Il-34 zur anwendung in einem verfahren zur behandlung von retinaler entzündung und neurodegeneration
US20210002667A1 (en) * 2018-03-15 2021-01-07 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
EP4169576A1 (de) 2018-03-23 2023-04-26 University of Massachusetts Gentherapeutika zur behandlung von knochenerkrankungen
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
EP3774852A1 (de) 2018-04-03 2021-02-17 Stridebio, Inc. Antikörpermeidende virale vektoren
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
CN112312929A (zh) 2018-04-18 2021-02-02 纽约市哥伦比亚大学理事会 用于包括线粒体dna耗竭综合征在内的由不平衡的核苷酸库引起的疾病的基因疗法
CA3098448A1 (en) 2018-04-27 2019-10-31 University Of Massachusetts Aav capsids identified by in vivo library selection
AU2019265663B2 (en) 2018-05-08 2024-06-13 Rutgers, The State University Of New Jersey AAV-compatible laminin-linker polymerization proteins
WO2019236139A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Interface for transporting ions from an atmospheric pressure environment to a low pressure environment
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
EP4391015A3 (de) 2018-06-04 2024-10-09 The Trustees of Indiana University Ionenfallenanordnung für hochdurchsatz-ladungserkennungsmassenspektrometrie
JP7398810B2 (ja) 2018-06-04 2023-12-15 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 静電線形イオン・トラップにイオンを捕獲する装置および方法
CA3100838A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Charge detection mass spectrometry with real time analysis and signal optimization
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
AU2019287468B2 (en) 2018-06-12 2024-10-10 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
EP3810782A2 (de) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vektoren zur genfreisetzung, die in zellen persistieren
BR112020025268A2 (pt) 2018-06-28 2021-03-09 The University Of North Carolina At Chapel Hill Cassetes de expressão e genes cln5 otimizados e seu uso
CN112654370A (zh) 2018-07-16 2021-04-13 西莱克塔生物科技公司 Mma构建体和载体的方法和组合物
WO2020018583A1 (en) 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
TW202020157A (zh) 2018-08-16 2020-06-01 美商艾爾妮蘭製藥公司 用於抑制lect2基因表現之組合物及方法
WO2020061528A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
US11965172B2 (en) 2018-11-05 2024-04-23 California Institute Of Technology DNA sequence modification-based gene drive
JP2022506066A (ja) 2018-11-05 2022-01-17 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 最適化されたｆｉｇ４遺伝子および発現カセットならびにそれらの使用
CN113574632B (zh) 2018-11-20 2024-07-30 印地安纳大学理事会 用于单粒子质谱分析的轨道阱
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
EP3887522A1 (de) 2018-11-29 2021-10-06 University of Massachusetts Modulation von sptlc1 über rekombinante adeno-assoziierte vektoren
EP4443473A3 (de) 2018-12-03 2025-01-01 The Trustees of Indiana University Vorrichtung zur gleichzeitigen analyse mehrerer ionen mit einer elektrostatischen linearen ionenfalle
SG11202106356QA (en) 2018-12-20 2021-07-29 Vigeneron Gmbh An optimized acceptor splice site module for biological and biotechnological applications
WO2020132385A1 (en) 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
US20220090094A1 (en) 2018-12-27 2022-03-24 Roche Molecular Systems, Inc. Non-replicative transduction particles and transduction particle-based reporter systems for detection of acinetobacter baumannii
US20220089670A1 (en) 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
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
CA3131023A1 (en) 2019-02-22 2020-08-27 Michael R. Volkert Oxr1 gene therapy
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Ｄｎａ結合ドメイントランスアクチベーター及びその使用
CN113795279B (zh) 2019-03-04 2024-12-31 宾夕法尼亚州大学信托人 靶向akt通路的神经保护性基因疗法
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
EP3952883A4 (de) 2019-04-12 2023-01-11 University of Massachusetts Gm3-synthasevektoren und deren verwendungen
CN114641318A (zh) 2019-04-15 2022-06-17 衣阿华大学研究基金会 用于治疗囊性纤维化的组合物和方法
BR112021020706A2 (pt) 2019-04-15 2022-03-15 Spirovant Sciences Inc Métodos e composições para expressão de transgene
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
SG11202111290WA (en) 2019-04-17 2021-11-29 Codiak Biosciences Inc Compositions of exosomes and aav
US20220211741A1 (en) 2019-04-18 2022-07-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment and prognosis of cancer
EP3959741A1 (de) 2019-04-23 2022-03-02 The Trustees of Indiana University Identifizierung von probensubspezies basierend auf partikelladungsverhalten unter strukturveränderungen induzierenden probenbedingungen
EP3959324B1 (de) 2019-04-26 2025-09-17 President and Fellows of Harvard College Aav-vektoren zur codierung von mini-pcdh15 und verwendungen davon
EP3962537A1 (de) 2019-04-28 2022-03-09 Selecta Biosciences, Inc. Verfahren zur behandlung von subjekten mit vorbestehender immunität gegen virusübertragungsvektoren
SG11202111375RA (en) 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
WO2020223215A1 (en) 2019-04-29 2020-11-05 The University Of North Carolina At Chapel Hill Optimized sumf1 genes and expression cassettes and their use
WO2020236815A1 (en) 2019-05-20 2020-11-26 University Of Massachusetts Minigene therapy
US12465655B2 (en) 2019-05-22 2025-11-11 The University Of North Carolina At Chapel Hill UBE3A genes and expression cassettes and their use
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
EP3987043A4 (de) * 2019-06-21 2023-07-19 Asklepios Biopharmaceutical, Inc. Herstellung von vektoren unter verwendung des phagenreplikationsursprungs
EP3990030A1 (de) 2019-06-27 2022-05-04 Pfizer Inc. Verfahren zur behandlung von duchenne-muskeldystrophie mittels aav-mini-dystrophin-gentherapie
US12492412B2 (en) 2019-07-02 2025-12-09 M6P Terapeutics (Switzerland) Llc Vector compositions and methods of using same for treatment of lysosomal storage disorders
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
EP4007814A1 (de) 2019-07-26 2022-06-08 Akouos, Inc. Verfahren zur behandlung von hörverlust unter verwendung eines sekretierten zielproteins
CN115176011B (zh) 2019-08-27 2025-11-11 赛诺菲 用于抑制pcsk9的组合物和方法
BR112022003860A2 (pt) 2019-09-03 2022-08-16 Alnylam Pharmaceuticals Inc Composições e métodos para inibir a expressão do gene lect2
AU2020346062A1 (en) 2019-09-13 2022-03-24 Rutgers, The State University Of New Jersey AAV-compatible laminin-linker polymerization proteins
KR20220128607A (ko) 2019-09-18 2022-09-21 인터갈락틱 테라퓨틱스, 인크. 합성 dna 벡터 및 사용 방법
WO2021062012A1 (en) 2019-09-25 2021-04-01 Emory University Use of klk10 and engineered derivatizations thereof
WO2021061650A1 (en) 2019-09-25 2021-04-01 The Trustees Of Indiana University Apparatus and method for pulsed mode charge detection mass spectrometry
US11492622B2 (en) 2019-09-26 2022-11-08 Massachusetts Institute Of Technology MicroRNA-based logic gates and uses thereof
WO2021062092A1 (en) 2019-09-26 2021-04-01 Massachusetts Institute Of Technology Trans-activated functional rna by strand displacement and uses thereof
US12503699B2 (en) 2019-10-04 2025-12-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing UGT1a1 gene expression
US20230416776A1 (en) 2019-10-08 2023-12-28 Regents Of The University Of Minnesota Crispr-mediated human genome editing with vectors
CN114728237B (zh) 2019-10-10 2026-02-24 印地安纳大学理事会 用于识别、选择和纯化粒子的系统和方法
WO2021076656A1 (en) 2019-10-15 2021-04-22 University Of Massachusetts Rna editor-enhanced rna trans-splicing
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
BR112022009216A2 (pt) 2019-11-13 2022-08-02 Alnylam Pharmaceuticals Inc Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt)
AU2020401116A1 (en) 2019-12-10 2022-07-21 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of Hunter disease
JP7690209B2 (ja) 2019-12-18 2025-06-10 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 電荷測定装置を有する質量分析計
WO2021136752A1 (en) 2019-12-31 2021-07-08 F. Hoffmann-La Roche Ag Quantitative pcr screening of inducible prophage from bacterial isolates
JP7703543B2 (ja) 2020-01-17 2025-07-07 ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド バイ ザ セクレタリー、デパートメント オブ ヘルス アンド ヒューマン サービシーズ Ｃｒｘ常染色体優性網膜症の処置のための遺伝子治療
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)
EP4100991B1 (de) 2020-02-03 2025-01-29 The Trustees of Indiana University Ein massenspektrometer zur ladungsdetektion und entsprechende methode
IL295445A (en) 2020-02-10 2022-10-01 Alnylam Pharmaceuticals Inc Preparations and methods for silencing vegf-a expression
US20230089312A1 (en) 2020-02-25 2023-03-23 University Of Massachusetts Inducible single aav system and uses thereof
US12605465B2 (en) 2020-03-23 2026-04-21 The University Of North Carolina At Chapel Hill AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021202494A1 (en) 2020-03-31 2021-10-07 University Of Massachusetts Aav capsids variants and uses thereof
KR20230008729A (ko) 2020-04-06 2023-01-16 알닐람 파마슈티칼스 인코포레이티드 Myoc 발현을 사일런싱하기 위한 조성물 및 방법
JP2023521094A (ja) 2020-04-07 2023-05-23 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 Ｓｃｎ９ａ発現をサイレンシングするための組成物および方法
CN115867650A (zh) 2020-04-20 2023-03-28 克里斯蒂安娜基因编辑研究公司 用于肺癌治疗的aav递送系统
JP2023524577A (ja) 2020-05-05 2023-06-12 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 改変アデノ随伴ウイルス５カプシドおよびその使用
CA3177869A1 (en) 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
IL298128A (en) 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
WO2021231538A2 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
US20230183707A1 (en) 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
WO2022006253A2 (en) 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors
WO2022011099A1 (en) 2020-07-08 2022-01-13 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
CA3186207A1 (en) 2020-07-23 2022-01-27 Rachel BAILEY Optimized slc13a5 genes and expression cassettes and their use
EP4196170A1 (de) 2020-08-14 2023-06-21 The Trustees of The University of Pennsylvania Neue aav-kapside und diese enthaltende zusammensetzungen
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CA3190399A1 (en) 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
WO2022047201A1 (en) 2020-08-27 2022-03-03 University Of Iowa Research Foundation Gene knock-out for treatment of glaucoma
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
EP4211232A1 (de) 2020-09-10 2023-07-19 Ludwig-Maximilians-Universität München Gentechnisch hergestellte aav-vektoren
EP4217376A4 (de) 2020-09-24 2024-10-09 University of Massachusetts Für nf1 codierende aav-vektoren und verwendungen davon
AU2021358781A1 (en) 2020-10-07 2023-05-18 Tern Therapeutics, Llc Gene therapy for ocular manifestations of cln2 disease
CA3195177A1 (en) 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)
WO2022079222A1 (en) 2020-10-16 2022-04-21 Sanofi Novel rna compositions and methods for inhibiting angptl3
WO2022079221A1 (en) 2020-10-16 2022-04-21 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
JP2023550581A (ja) 2020-10-29 2023-12-04 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ａａｖカプシド及びそれを含有する組成物
TW202237150A (zh) 2020-12-01 2022-10-01 美商艾拉倫製藥股份有限公司 用於抑制ｈａｏ１（羥基酸氧化酶１（乙醇酸氧化酶））基因表現的方法及組成物
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
KR20230117177A (ko) 2020-12-03 2023-08-07 유니버시티 오브 매사추세츠 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발
IL304006A (en) 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1
WO2022150335A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向ｔ細胞之ａａｖ載體
EP4281559A4 (de) 2021-01-22 2024-12-04 University of Massachusetts Verwendung von neuen mirna-bindungsstellenkassetten zur detargetierung antigenpräsentierender zellen durch vektoren der raav-gentherapie
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022170082A1 (en) 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
AR124981A1 (es) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
CN113549642B (zh) * 2021-03-02 2023-03-07 体必康生物科技(广东)股份有限公司 一种结核分枝杆菌整合型表达质粒及其应用
US11760788B2 (en) 2021-03-02 2023-09-19 Pathways Neuro Pharma, Inc. Neuroreceptor compositions and methods of use
WO2022198038A1 (en) 2021-03-19 2022-09-22 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
WO2022211791A1 (en) 2021-03-30 2022-10-06 AAVnerGene Inc. Modified plasma clotting factor viii and method of use thereof
US11795207B2 (en) 2021-03-30 2023-10-24 AAVnerGene Inc. Modified plasma clotting factor VIII and method of use thereof
WO2022221684A1 (en) 2021-04-15 2022-10-20 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
WO2022221529A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
US20240181084A1 (en) 2021-04-23 2024-06-06 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas Fusion Protein and Methods of Treatment
WO2022232041A1 (en) 2021-04-26 2022-11-03 University Of Massachusetts Gene therapies for stargardt disease (abca4)
WO2022232267A1 (en) 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
WO2022258755A1 (en) 2021-06-09 2022-12-15 Universität Duisburg-Essen Method for immortalising vesicle-secreting cells
CN117715928A (zh) 2021-06-14 2024-03-15 桥生物基因疗法研究有限责任公司 用于结节性硬化的基因疗法
AU2022303164A1 (en) 2021-06-30 2024-01-18 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2023279108A1 (en) 2021-07-01 2023-01-05 Bridgebio Gene Therapy Research, Inc. Gene therapy for galactosemia
WO2023004370A1 (en) 2021-07-21 2023-01-26 Aspen Neuroscience, Inc. Aav-based modulation of gba1 and related compositions and uses thereof
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
JP2024538859A (ja) 2021-08-31 2024-10-24 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 細胞死誘導ＤＦＦＡ様エフェクターＢ（ＣＩＤＥＢ）ｉＲＮＡ組成物およびその使用方法
JP2024537775A (ja) 2021-09-30 2024-10-16 アコーオス インコーポレイテッド Ｋｃｎｑ４関連難聴を治療するための組成物及び方法
AU2022366947A1 (en) 2021-10-12 2024-05-02 Bridgebio Gene Therapy Llc Methods and compositions for treating leukodystrophies
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023064350A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Viral vector dosing protocols
JP2024545594A (ja) 2021-11-14 2024-12-10 カーティザン セラピューティクス，インコーポレーテッド ウイルスベクターでの複数回投薬
US20250011812A1 (en) 2021-11-23 2025-01-09 University Of Massachusetts Gene therapy for spinal muscular atrophy
CA3239342A1 (en) 2021-11-30 2023-06-08 Michael Hughes Gene therapy methods for treatment of diabetes
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
JP2024545923A (ja) 2021-12-30 2024-12-13 リーゲル セラピューティクス，インコーポレイティッド ナトリウム電位依存性チャネルアルファサブユニット１の発現を調節するための組成物、及びその使用
WO2023147383A1 (en) 2022-01-25 2023-08-03 Bridgebio Services Inc. Gene therapy for genetic hearing loss
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
TW202342525A (zh) 2022-02-02 2023-11-01 美商阿科奧斯公司 用於治療前庭神經鞘瘤相關症狀之抗ｖｅｇｆ抗體構築體及相關方法
TW202342759A (zh) 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
US20250179531A1 (en) 2022-02-25 2025-06-05 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
CN119301262A (zh) 2022-04-01 2025-01-10 武田药品工业株式会社 用于具有cns表现的疾病的基因疗法
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
CN119403933A (zh) 2022-06-21 2025-02-07 里格尔医疗股份有限公司 遗传调控元件及其用途
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
CA3262218A1 (en) 2022-07-19 2024-01-25 Rampart Bioscience, Inc. CIRCULAR NON-IMMUNOGENEOUS NON-VIRAL DNA VECTORS
CN120813689A (zh) 2022-08-11 2025-10-17 笛卡尔疗法股份有限公司 与免疫球蛋白蛋白酶及其融合体相关的组合物和方法
WO2024047587A1 (en) 2022-08-31 2024-03-07 Regel Therapeutics, Inc. Cas-phi compositions and methods of use
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024092171A1 (en) 2022-10-26 2024-05-02 University Of Iowa Research Foundation Method to deliver large genes using virus and a dna recombination system
WO2024098061A2 (en) 2022-11-04 2024-05-10 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
EP4619524A1 (de) 2022-11-18 2025-09-24 Genkardia Inc. Verfahren und zusammensetzungen zur prävention, behandlung oder umkehrung von diastolischer herzdysfunktion
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2024129696A1 (en) 2022-12-12 2024-06-20 Retromer Therapeutics Corp. Aav and lentiviral constructs comprising a sorl1 mini-gene for use in treating neurodegenerative diseases
IL322049A (en) 2023-02-01 2025-09-01 Sarepta Therapeutics Inc Methods for producing raav
WO2024178386A1 (en) 2023-02-24 2024-08-29 Aarhus Universitet Methods of treating endosomal trafficking diseases
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
KR20260010739A (ko) 2023-05-11 2026-01-21 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 항불안 치료
WO2024233791A1 (en) 2023-05-11 2024-11-14 Seelos Therapeutics, Inc. Methods of treating neurodegenerative disorders
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024249172A1 (en) 2023-05-26 2024-12-05 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 2 and uses thereof
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025083591A2 (en) 2023-10-17 2025-04-24 Regel Therapeutics, Inc. Genetic regulatory elements for cardiac tissue expression and uses thereof
WO2025117745A1 (en) 2023-12-01 2025-06-05 Caribou Biosciences, Inc. Methods and compositions for improving precision of endonuclease-mediated genome editing
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025227063A1 (en) 2024-04-26 2025-10-30 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025235491A1 (en) 2024-05-07 2025-11-13 Affinia Therapeutics Inc. Recombinant aav for treatment of cardiac diseases
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
WO2026020116A1 (en) 2024-07-18 2026-01-22 Massachusetts Institute Of Technology Methods and compositions for enriching the production of dna cargo loaded recombinant adeno-associated virus capsids
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064739A1 (en) 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2026064736A1 (en) 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy

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
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1991018088A1 (en) * 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors

Also Published As

Publication number Publication date
US5478745A (en) 1995-12-26
EP0673415B1 (de) 2009-02-18
IL107862A0 (en) 1994-04-12
JP4117023B2 (ja) 2008-07-09
KR100256890B1 (ko) 2000-05-15
DE69334262D1 (de) 2009-04-02
TW260708B (de) 1995-10-21
JPH08504102A (ja) 1996-05-07
EP0673415A4 (de) 1997-05-21
WO1994013788A1 (en) 1994-06-23
EP0673415A1 (de) 1995-09-27

Similar Documents

Publication Publication Date Title
ATE423203T1 (de) Rekombinantes virales vektorsystem
ATE411818T1 (de) Rekombinantes,virales vektorsystem
PT836648E (pt) Virus mva recombinante e sua utilizacao
EP1005376A4 (de) Methoden und zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie
ATE470704T1 (de) Rekombinantes sendai-virus
DE3853139D1 (de) Rekombinanter, fremde gene enthaltender menschlicher cytomegalovirus und seine verwendung.
NO964874L (no) Fusjonsglykoprotein fra HCMV og HSV
FR2724320B1 (fr) Nouvel implant pour le traitement des maladies acquises
HUP9802587A2 (hu) Glükózzal indukálható rekombináns virális vektor, ezt tartalmazó gyógyszerkészítmény és ezzel transzformált sejtek
CA2158869A1 (fr) Virus recombinants, preparation et utilisation en therapie genique
MY119381A (en) Recombinant mva virus, and the use thereof
MY134484A (en) Recombinant mva virus, and the use thereof

Legal Events

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