ATE217904T1 - Nukleozyme - Google Patents

Nukleozyme

Info

Publication number
ATE217904T1
ATE217904T1 AT93904752T AT93904752T ATE217904T1 AT E217904 T1 ATE217904 T1 AT E217904T1 AT 93904752 T AT93904752 T AT 93904752T AT 93904752 T AT93904752 T AT 93904752T AT E217904 T1 ATE217904 T1 AT E217904T1
Authority
AT
Austria
Prior art keywords
nucleozymes
degradation
resistant
nucleic acid
further comprise
Prior art date
Application number
AT93904752T
Other languages
English (en)
Inventor
Nassim Usman
Robert J Cedergren
Jean-Pierre Perreault
Jing-Hua Yang
Alexander Rich
Original Assignee
Massachusetts Inst Technology
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=25255389&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ATE217904(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Massachusetts Inst Technology filed Critical Massachusetts Inst Technology
Application granted granted Critical
Publication of ATE217904T1 publication Critical patent/ATE217904T1/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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Communicable Diseases (AREA)
  • AIDS & HIV (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AT93904752T 1992-01-31 1993-01-28 Nukleozyme ATE217904T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US07/829,729 US5652094A (en) 1992-01-31 1992-01-31 Nucleozymes
PCT/US1993/000833 WO1993015187A1 (en) 1992-01-31 1993-01-28 Nucleozymes

Publications (1)

Publication Number Publication Date
ATE217904T1 true ATE217904T1 (de) 2002-06-15

Family

ID=25255389

Family Applications (1)

Application Number Title Priority Date Filing Date
AT93904752T ATE217904T1 (de) 1992-01-31 1993-01-28 Nukleozyme

Country Status (9)

Country Link
US (4) US5652094A (de)
EP (3) EP1162263A1 (de)
JP (1) JPH07503613A (de)
AT (1) ATE217904T1 (de)
AU (1) AU680405B2 (de)
CA (1) CA2129119A1 (de)
DE (1) DE69331946T2 (de)
MX (1) MX9300499A (de)
WO (1) WO1993015187A1 (de)

Families Citing this family (303)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA1223831A (en) 1982-06-23 1987-07-07 Dean Engelhardt Modified nucleotides, methods of preparing and utilizing and compositions containing the same
US5646042A (en) * 1992-08-26 1997-07-08 Ribozyme Pharmaceuticals, Inc. C-myb targeted ribozymes
US5891684A (en) * 1992-10-15 1999-04-06 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5811300A (en) * 1992-12-07 1998-09-22 Ribozyme Pharmaceuticals, Inc. TNF-α ribozymes
US5616488A (en) * 1992-12-07 1997-04-01 Ribozyme Pharmaceuticals, Inc. IL-5 targeted ribozymes
US5612215A (en) * 1992-12-07 1997-03-18 Ribozyme Pharmaceuticals, Inc. Stromelysin targeted ribozymes
US5837542A (en) 1992-12-07 1998-11-17 Ribozyme Pharmaceuticals, Inc. Intercellular adhesion molecule-1 (ICAM-1) ribozymes
US5658780A (en) * 1992-12-07 1997-08-19 Ribozyme Pharmaceuticals, Inc. Rel a targeted ribozymes
ATE227342T1 (de) * 1993-09-02 2002-11-15 Ribozyme Pharm Inc Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
WO1995006764A2 (en) * 1993-09-03 1995-03-09 Vpi Holdings Ltd. Oligonucleotides with rna cleavage activity
US5861288A (en) * 1993-10-18 1999-01-19 Ribozyme Pharmaceuticals, Inc. Catalytic DNA
ATE174600T1 (de) * 1993-10-27 1999-01-15 Ribozyme Pharm Inc 2'-amido-und 2'-peptido-modifizierte oligonukleotide
CA2176035A1 (en) * 1993-11-08 1995-05-18 Nassim Usman Base-modified enzymatic nucleic acid
US5631359A (en) * 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5693532A (en) * 1994-11-04 1997-12-02 Ribozyme Pharmaceuticals, Inc. Respiratory syncytial virus ribozymes
US5639647A (en) * 1994-03-29 1997-06-17 Ribozyme Pharmaceuticals, Inc. 2'-deoxy-2'alkylnucleotide containing nucleic acid
US5670347A (en) 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
US6103890A (en) * 1994-05-18 2000-08-15 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids that cleave C-fos
US5633133A (en) * 1994-07-14 1997-05-27 Long; David M. Ligation with hammerhead ribozymes
US5599706A (en) * 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US5700923A (en) * 1994-09-29 1997-12-23 Hybridon, Inc. Finderons and methods of their preparation and use
US5650502A (en) * 1994-11-09 1997-07-22 Hybridon, Inc. Ribozyme analogs having rigid non-nucleotidic linkers
US5716824A (en) * 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5672501A (en) * 1994-12-23 1997-09-30 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5545729A (en) * 1994-12-22 1996-08-13 Hybridon, Inc. Stabilized ribozyme analogs
US5705388A (en) * 1994-12-23 1998-01-06 Ribozyme Pharmaceuticals, Inc. CETP Ribozymes
US5663064A (en) * 1995-01-13 1997-09-02 University Of Vermont Ribozymes with RNA protein binding site
US5877021A (en) * 1995-07-07 1999-03-02 Ribozyme Pharmaceuticals, Inc. B7-1 targeted ribozymes
US20030207837A1 (en) * 1995-07-07 2003-11-06 Ribozyme Pharmaceuticals, Inc. Method and reagent for the induction of graft tolerance and reversal of immune responses
US6346398B1 (en) 1995-10-26 2002-02-12 Ribozyme Pharmaceuticals, Inc. Method and reagent for the treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
WO1997018312A1 (en) * 1995-11-14 1997-05-22 Vimrx Holdings, Ltd. Chimeric oligomers having an rna-cleavage activity
US5998203A (en) * 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6274369B1 (en) * 1996-02-02 2001-08-14 Invitrogen Corporation Method capable of increasing competency of bacterial cell transformation
US5807743A (en) * 1996-12-03 1998-09-15 Ribozyme Pharmaceuticals, Inc. Interleukin-2 receptor gamma-chain ribozymes
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
ATE332368T1 (de) * 1997-01-21 2006-07-15 Gen Hospital Corp Selektion von proteinen mittels rns-protein fusionen
US6057156A (en) 1997-01-31 2000-05-02 Robozyme Pharmaceuticals, Inc. Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6159951A (en) 1997-02-13 2000-12-12 Ribozyme Pharmaceuticals Inc. 2'-O-amino-containing nucleoside analogs and polynucleotides
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
US5942395A (en) * 1997-05-09 1999-08-24 Universite De Montreal Hybrid ribozymes and methods of use
US6548657B1 (en) 1997-06-09 2003-04-15 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6280936B1 (en) 1997-06-09 2001-08-28 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6183959B1 (en) 1997-07-03 2001-02-06 Ribozyme Pharmaceuticals, Inc. Method for target site selection and discovery
US6316612B1 (en) 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
JP2001518292A (ja) * 1997-09-22 2001-10-16 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ エンドヌクレアーゼ活性を有する核酸触媒
US6656731B1 (en) 1997-09-22 2003-12-02 Max Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
US6054576A (en) * 1997-10-02 2000-04-25 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA
US6127535A (en) 1997-11-05 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into oligonucleotides
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US20020147143A1 (en) 1998-03-18 2002-10-10 Corixa Corporation Compositions and methods for the therapy and diagnosis of lung cancer
US6307041B1 (en) 1998-03-28 2001-10-23 University Of Utah Research Foundation Circular, hairpin, circular/hairpin, lariat, and hairpin-lariat hammerhead ribozymes
EP1071753A2 (de) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nukeinsäure moleküle mit einer neuen chemischen zusammensetzung die fähig sind die expression von gene zu modulieren
US6995259B1 (en) 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
EP1183389A2 (de) 1999-04-30 2002-03-06 University of Florida Ribozym zusamensetzungen verabreicht durch adeno asozierte viren und dessen verwendungen
US6831171B2 (en) 2000-02-08 2004-12-14 Yale University Nucleic acid catalysts with endonuclease activity
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US20040002068A1 (en) 2000-03-01 2004-01-01 Corixa Corporation Compositions and methods for the detection, diagnosis and therapy of hematological malignancies
AR029540A1 (es) 2000-06-28 2003-07-02 Corixa Corp COMPOSICIONES Y METODOS PARA EL DIAGNoSTICO Y LA TERAPIA DE CA NCER DE PULMoN
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
US7125660B2 (en) * 2000-09-13 2006-10-24 Archemix Corp. Nucleic acid sensor molecules and methods of using same
WO2002022882A2 (en) * 2000-09-13 2002-03-21 Archemix Corporation Target activated nucleic acid biosensor and methods of using same
EP1326892A2 (de) 2000-10-12 2003-07-16 University of Rochester Zusammensetzungen die die proliferation von krebszellen hemmen
WO2002072790A2 (en) 2001-03-14 2002-09-19 Myriad Genetics, Inc Tsg101-gag interaction and use thereof
EP1386004A4 (de) 2001-04-05 2005-02-16 Ribozyme Pharm Inc Modulation der mit der entzündungsausbreitung und dem neuritenauswuchs assoziierten genexpression unter verwendung von technologien auf nukleinsäurebasis
JP2005504513A (ja) 2001-05-09 2005-02-17 コリクサ コーポレイション 前立腺癌の治療及び診断のための組成物及び方法
US7517864B2 (en) 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
EP2415486B1 (de) 2001-05-18 2017-02-22 Sirna Therapeutics, Inc. Konjugate und Zusammensetzungen zur zellulären Verabreichung
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7205399B1 (en) 2001-07-06 2007-04-17 Sirna Therapeutics, Inc. Methods and reagents for oligonucleotide synthesis
DK2224012T3 (da) 2001-12-17 2013-05-13 Corixa Corp Sammensætninger og fremgangsmåder til terapi og diagnose af inflammatoriske tarmsygdomme
WO2003065997A2 (en) 2002-02-06 2003-08-14 Vicor Technologies, Inc. Anti-infarction molecules
US20050042632A1 (en) * 2002-02-13 2005-02-24 Sirna Therapeutics, Inc. Antibodies having specificity for nucleic acids
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1432724A4 (de) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Durch rna-interferenz vermittelte inhibierung von map-kinase-genen
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003253580A1 (en) * 2002-02-26 2003-11-17 University Of Minnesota Variants of nedd4l associated with hypertension and viral budding
US20030195164A1 (en) * 2002-04-16 2003-10-16 Veli-Matti Kahari Novel ribozyme and its use
US7655790B2 (en) 2002-07-12 2010-02-02 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US6989442B2 (en) 2002-07-12 2006-01-24 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
EP1575992A4 (de) 2002-08-05 2007-02-21 Univ Rochester Chimäre proteine aus proteintransduzierender domäne/deaminase, verwandte verbindungen und deren verwendungen
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20080260744A1 (en) 2002-09-09 2008-10-23 Omeros Corporation G protein coupled receptors and uses thereof
AU2003298921A1 (en) * 2002-12-04 2004-06-23 Algos Therapeutics, Inc. Methods and materials for modulating trpm2
US7785782B2 (en) * 2002-12-12 2010-08-31 Novartis Vaccines And Diagnostics, Inc. Device and method for in-line blood testing using biochips
EP3222294A1 (de) 2003-04-30 2017-09-27 Sirna Therapeutics, Inc. Konjugate und zusammensetzungen zur zellulären verabreichung
JP2005017116A (ja) * 2003-06-26 2005-01-20 Sharp Corp 光学式エンコーダ用受光素子
US20060019914A1 (en) 2004-02-11 2006-01-26 University Of Tennessee Research Foundation Inhibition of tumor growth and invasion by anti-matrix metalloproteinase DNAzymes
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
EP1747022A4 (de) 2004-04-23 2010-03-31 Univ Columbia Hemmung von haarloser protein mrna
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
WO2006020684A2 (en) 2004-08-10 2006-02-23 Institute For Multiple Myeloma And Bone Cancer Research Methods of regulating differentiation and treating of multiple myeloma
US7568565B2 (en) * 2004-08-17 2009-08-04 Nes Technologies, Inc Device, a system and a method for transferring vibrational energy
ES2401285T3 (es) 2004-12-16 2013-04-18 The Regents Of The University Of California Fármacos con el pulmón como diana
JP5562521B2 (ja) 2005-02-02 2014-07-30 ザ ユーエービー リサーチ ファンデーション アポトーシス誘導デスレセプターアゴニストに対する抵抗性を低減することに関する薬剤及び方法
JP5225069B2 (ja) 2005-03-23 2013-07-03 ゲンマブ エー／エス 多発性骨髄腫の治療のためのｃｄ３８に対する抗体
US7476733B2 (en) 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
JP2008537551A (ja) 2005-03-31 2008-09-18 カランド ファーマシューティカルズ， インコーポレイテッド リボヌクレオチドレダクターゼサブユニット２の阻害剤およびその使用
WO2007002904A2 (en) 2005-06-28 2007-01-04 Medtronic, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
WO2007044607A2 (en) * 2005-10-06 2007-04-19 Emthrax, Llc Methods and compositions relating to anthrax spore glycoproteins as vaccines
EP2392645A1 (de) 2005-10-14 2011-12-07 MUSC Foundation For Research Development Targeting von PAX2 zur Induktion der DEFB1-vermittelten Tumorimmunität und Krebstherapie
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
PT1981902E (pt) 2006-01-27 2015-11-02 Biogen Ma Inc Antagonistas dos recetores nogo
NZ571568A (en) 2006-03-31 2010-11-26 Alnylam Pharmaceuticals Inc Double-stranded RNA molecule compositions and methods for inhibiting expression of Eg5 gene
WO2007117657A2 (en) 2006-04-07 2007-10-18 The Research Foundation Of State University Of New York Transcobalamin receptor polypeptides, nucleic acids, and modulators thereof, and related methods of use in modulating cell growth and treating cancer and cobalamin deficiency
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
ES2479668T3 (es) 2006-07-11 2014-07-24 University Of Medicine And Dentistry Of New Jersey Proteínas de reparación de la membrana celular, ácidos nucleicos que las codifican y métodos de uso asociados
EP2560001B1 (de) 2006-09-21 2016-04-13 University of Rochester Zusammensetzungen und Verfahren für eine Proteinverlagerungstherapie für myotone Dystrophie
EP2099467B1 (de) 2006-10-03 2017-05-10 University Of Medicine And Dentistry Of New Jersey Atap-proteine, für diese kodierende nukleinsäuren und entsprechende verwendungsverfahren
WO2008067040A2 (en) 2006-10-06 2008-06-05 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
WO2008052774A2 (en) 2006-10-31 2008-05-08 Noxxon Pharma Ag Methods for detection of a single- or double-stranded nucleic acid molecule
US8999317B2 (en) 2006-11-01 2015-04-07 University Of Rochester Methods and compositions related to the structure and function of APOBEC3G
BRPI0720038A2 (pt) 2006-12-11 2013-12-24 Univ Utah Res Found Métodos para inibir a permeabilidade vascular em tecido, para triar ou avaliar um agente que inibe a permeabilidade vascular, para tratar ou prevenir a síndrome da angústia respiratória, a retinopatia de prematuridade, a retinopatia diabética e a degeneração macular úmida em um indivíduo, para tratar indivíduos com sugestões repulsivas ou miméticos e para promover a angiogênese em um tecido, polipeptídeo isolado, ácido nucleico isolado, e, vetor
US20100129358A1 (en) 2006-12-22 2010-05-27 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
HUE040417T2 (hu) 2007-05-04 2019-03-28 Marina Biotech Inc Aminosavlipidek és alkalmazásuk
EP2195331B1 (de) 2007-08-28 2013-11-20 Uab Research Foundation Synthetische, apolipoprotein e nachahmende polypeptide und anwendungsverfahren
WO2009032702A2 (en) 2007-08-28 2009-03-12 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
WO2009033027A2 (en) 2007-09-05 2009-03-12 Medtronic, Inc. Suppression of scn9a gene expression and/or function for the treatment of pain
WO2010070380A2 (en) 2007-12-03 2010-06-24 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health Of Human Services, National Institutes Of Health Doc1 compositions and methods for treating cancer
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
US20090233993A1 (en) * 2008-03-06 2009-09-17 Burnham Institute For Medical Research Compositions and methods for inhibiting gsk3 activity and uses thereof
AU2009232355A1 (en) 2008-04-04 2009-10-08 Calando Pharmaceuticals, Inc. Compositions and use of EPAS1 inhibitors
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US20090274696A1 (en) * 2008-04-29 2009-11-05 Wyeth Methods for treating inflammation
US20110262395A1 (en) 2008-05-08 2011-10-27 University Of Utah Research Foundation Sensory receptors for chronic fatigue and pain and uses thereof
WO2009155100A1 (en) 2008-05-30 2009-12-23 Yale University Targeted oligonucleotide compositions for modifying gene expression
EP2323679A4 (de) 2008-07-25 2012-08-22 Univ Colorado Clip-hemmer und verfahren zur modulierung der immunfunktion
EP3025727A1 (de) 2008-10-02 2016-06-01 The J. David Gladstone Institutes Verfahren zur behandlung von lebererkrankungen
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
JP2012505913A (ja) 2008-10-16 2012-03-08 マリーナ バイオテック，インコーポレイテッド 遺伝子発現を抑制する治療におけるリポソームによる効率的な送達のプロセスおよび組成物
EP2346904B1 (de) 2008-10-29 2017-04-12 China Synthetic Rubber Corporation Methoden und mittel zur diagnose und behandlung von hepatozellulärem karzinom
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
US20120070443A1 (en) 2008-12-02 2012-03-22 University Of Utah Research Foundation Pde1 as a target therapeutic in heart disease
EP3243504A1 (de) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Verbesserte lipidformulierung
US20100215660A1 (en) 2009-02-23 2010-08-26 Sarwar Hashmi Kruppel-like factors and fat regulation
EP2756845B1 (de) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Verfahren und Zusammensetzungen für spezifische KRAS-Hemmung mit asymmetrischer Doppelstrang-RNA
CN103555722B (zh) 2009-04-03 2016-08-31 戴瑟纳制药公司 利用不对称双链rna特异性抑制kras的方法和组合物
CA2758189C (en) 2009-04-10 2020-12-29 Association Institut De Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
CN107252489A (zh) 2009-04-13 2017-10-17 法国健康和医学研究院 Hpv颗粒及其用途
CA3045126A1 (en) 2009-05-05 2010-11-11 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
CA2762095A1 (en) 2009-05-16 2010-11-25 Kunyuan Cui Compositions comprising cationic amphiphiles and colipids for delivering therapeutic molecules
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011031974A1 (en) 2009-09-10 2011-03-17 Southern Research Institute Acridine analogs in the treatment of gliomas
WO2011039646A2 (en) 2009-09-30 2011-04-07 Inserm (Institut National De La Sante Et De La Recherche Medicale) Papilloma virus -like particles for targeted gene delivery
US8450090B2 (en) 2009-10-06 2013-05-28 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for promoting fatty acid production in plants
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
EP3296398A1 (de) 2009-12-07 2018-03-21 Arbutus Biopharma Corporation Zusammensetzungen für nukleinsäurefreisetzung
EP3494963A1 (de) 2009-12-18 2019-06-12 The University of British Columbia Verfahren und zusammensetzungen zur abgabe von nukleinsäuren
US20120296403A1 (en) 2010-02-10 2012-11-22 Novartis Ag Methods and compounds for muscle growth
EP2516681B1 (de) 2010-02-11 2017-10-18 Nanostring Technologies, Inc Zusammensetzungen und verfahren zur erkennung von vorzugsweise kleiner rnas mittels brücken hybridisierung und ligation
US20110207789A1 (en) 2010-02-19 2011-08-25 Ye Fang Methods related to casein kinase ii (ck2) inhibitors and the use of purinosome-disrupting ck2 inhibitors for anti-cancer therapy agents
JP2013529181A (ja) 2010-03-25 2013-07-18 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 神経障害を治療するための組成物および方法
WO2011120023A1 (en) 2010-03-26 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting survivin gene expression uses thereof
WO2011133584A2 (en) 2010-04-19 2011-10-27 Marina Biotech, Inc. Nucleic acid compounds for inhibiting hras gene expression and uses thereof
SG10201800541SA (en) 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
EP2563922A1 (de) 2010-04-26 2013-03-06 Marina Biotech, Inc. Nukleinsäureverbindungen mit konformationseingeschränkten monomeren und ihre verwendung
WO2011139843A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Multi-sirna compositions for reducing gene expression
AU2011276365B2 (en) 2010-07-06 2017-05-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of beta-catenin by double-stranded RNA
US10064885B2 (en) 2010-07-09 2018-09-04 Massachusetts Institute Of Technology Metabolic gene, enzyme, and flux targets for cancer therapy
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US9243246B2 (en) 2010-08-24 2016-01-26 Sirna Therapeutics, Inc. Single-stranded RNAi agents containing an internal, non-nucleic acid spacer
WO2012044992A2 (en) 2010-09-30 2012-04-05 Agency For Science, Technology And Research (A*Star) Methods and reagents for detection and treatment of esophageal metaplasia
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
WO2012068463A2 (en) 2010-11-18 2012-05-24 Beth Israel Deaconess Medicall Center, Inc. Methods of treating obesity by inhibiting nicotinamide n-methyl transferase (nnmt)
AU2012205583B2 (en) 2011-01-11 2016-01-21 Fate Therapeutics, Inc. Novel Wnt compositions and therapeutic uses of such compositions
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP2753631A1 (de) 2011-09-07 2014-07-16 Marina Biotech, Inc. Synthese und verwendung von nukleinsäureverbindungen mit konformationseingeschränkten monomeren
EP3456317B1 (de) 2011-09-27 2025-09-24 Alnylam Pharmaceuticals, Inc. Substituierte di-aliphatic pegylierte lipide
BR112014008862A2 (pt) 2011-10-14 2018-08-07 Genentech Inc anticorpo isolado que se liga à htra1, ácido nucleico isolado, célula hospedeira, imunoconjugado, formulação farmacêutica, métodos e usos
US20150291999A1 (en) 2011-10-14 2015-10-15 Accugenomics, Inc. Nucleic acid amplification and use thereof
JP2015502365A (ja) 2011-12-12 2015-01-22 オンコイミューニン，インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
EP2797948B1 (de) 2011-12-27 2019-02-20 The J. David Gladstone Institutes Zusammensetzungen zur verwendung in der regulierung des glukosemetabolismus
WO2013119877A1 (en) 2012-02-07 2013-08-15 Aura Biosciences, Inc. Virion-derived nanospheres for selective delivery of therapeutic and diagnostic agents to cancer cells
EP2817287B1 (de) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Kationische trialkyllipide und verfahren zur verwendung davon
EP2855704A4 (de) 2012-05-25 2016-03-16 Accugenomics Inc Nukleinsäureamplifikation und anwendung davon
KR101520383B1 (ko) 2012-08-02 2015-05-15 에이비온 주식회사 Ｈｐｖ 감염과 관련된 암의 치료용 조성물
SG10201800188SA (en) 2013-03-15 2018-02-27 Univ California Acyclic nucleoside phosphonate diesters
US9458457B2 (en) 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
WO2015006740A2 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
EP3046583B1 (de) 2013-09-18 2019-02-13 Aura Biosciences, Inc. Virusähnliche partikelkonjugate zur behandlung von tumoren
WO2015048531A1 (en) 2013-09-26 2015-04-02 Beth Israel Deaconess Medical Center, Inc. Inhibition of sgk1 in the treatment of heart conditions
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10772974B2 (en) 2013-11-18 2020-09-15 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for cardiac regeneration
CN106061488B (zh) 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3084446B1 (de) 2013-12-20 2022-12-14 Acetylon Pharmaceuticals, Inc. Histondeacetylase 6 (hdac6)-biomarker bei multiplem myelom
EP3581654B1 (de) 2013-12-27 2021-03-31 Dicerna Pharmaceuticals, Inc. Verfahren und zusammensetzungen für die spezifische hemmung von glycolatoxidase (hao1) durch doppelstrang-rna
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016018665A1 (en) 2014-07-31 2016-02-04 Uab Research Foundation Apoe mimetic peptides and higher potency to clear plasma cholesterol
CN111808136B (zh) 2014-09-15 2024-06-11 加利福尼亚大学董事会 核苷酸类似物
AU2015330670B2 (en) 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
WO2016061131A1 (en) 2014-10-14 2016-04-21 The J. David Gladstone Institutes Compositions and methods for reactivating latent immunodeficiency virus
JP6637988B2 (ja) 2014-11-18 2020-01-29 アークトゥルス セラピューティクス， インコーポレイテッド Ｒｎａ送達のためのイオン化可能カチオン性脂質
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
EP3331536A4 (de) 2015-08-03 2019-03-27 The Regents of The University of California Zusammensetzungen und verfahren zur modulierung der abhd2-aktivität
EP3350191B9 (de) 2015-09-15 2021-12-22 The Regents of the University of California Nukleotidanaloga
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
MY196448A (en) 2015-10-30 2023-04-12 Genentech Inc Anti-Htra1 Antibodies and Methods of use Thereof
JP2018532801A (ja) 2015-10-30 2018-11-08 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド 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 標的化がん療法
WO2017173297A1 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Beta-catenin nucleic acids and uses thereof
WO2017173301A1 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Egfr nucleic acids and uses thereof
WO2017173320A2 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Myc nucleic acids and uses thereof
EP3436585B1 (de) 2016-04-01 2022-07-20 Avidity Biosciences, Inc. Kras-nukleinsäuren und verwendungen davon
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
US20170283795A1 (en) 2016-04-01 2017-10-05 Avidity Biosciences Llc Phosphatidylinositol-3-kinase nucleic acids and uses thereof
WO2017205506A1 (en) 2016-05-24 2017-11-30 Emory University Particles with rna cleaving nucleobase polymers and uses for managing inflammatory disorders
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
CN110087665A (zh) 2016-08-03 2019-08-02 H·李·莫菲特癌症中心与研究所公司 Tlr9靶向治疗
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
MA45996A (fr) 2016-08-16 2021-06-02 Bluebird Bio Inc Variants d'endonucléase de homing du récepteur alpha de l'il-10, compositions et méthodes d'utilisation associées
WO2018039333A1 (en) 2016-08-23 2018-03-01 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102451510B1 (ko) 2016-09-08 2022-10-07 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
US11591582B2 (en) 2016-10-11 2023-02-28 2Seventy Bio, Inc. TCRα homing endonuclease variants
MA46862A (fr) 2016-11-17 2019-09-25 Bluebird Bio Inc Convertisseur de signaux tgf béta
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119163A1 (en) 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
EP3583203B1 (de) 2017-02-15 2023-11-01 2seventy bio, Inc. Spenderreparaturtemplates für multiplex-genomeditierung
EP3592853A1 (de) 2017-03-09 2020-01-15 President and Fellows of Harvard College Unterdrückung von schmerzen durch geneditierung
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
IL269986B2 (en) 2017-04-12 2024-06-01 Aura Biosciences Inc Targeted combination therapy
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (de) 2017-08-30 2025-01-15 President and Fellows of Harvard College Hocheffiziente baseneditoren mit gam
WO2019046698A1 (en) 2017-09-01 2019-03-07 Thomas Jefferson University COMPOSITIONS AND METHODS FOR MYC GENE MESSENGER RNA INHIBITORS
US12016314B2 (en) 2017-09-08 2024-06-25 Ohio State Innovation Foundation MicroRNA inhibitor therapy in systemic lupus erythematosus
EP3691657A4 (de) 2017-10-04 2021-07-21 Avidity Biosciences, Inc. Nukleinsäurepolypeptidzusammensetzungen und verwendungen davon
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
AU2018378812B2 (en) 2017-12-06 2025-05-22 Avidity Biosciences, Inc. Compositions and methods of treating muscle atrophy and myotonic dystrophy
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
AU2019394996B2 (en) 2018-12-06 2025-11-06 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
KR20210104772A (ko) 2018-12-14 2021-08-25 블루버드 바이오, 인코포레이티드. 이량체화제로 조절된 면역수용체 복합체
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220204975A1 (en) 2019-04-12 2022-06-30 President And Fellows Of Harvard College System for genome editing
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
CN114375296A (zh) 2019-06-06 2022-04-19 艾维迪提生物科学公司 核酸多肽组合物及其用途
US12006499B2 (en) 2019-06-06 2024-06-11 Avidity Biosciences, Inc. Una amidites and uses thereof
CN114901253A (zh) 2019-08-14 2022-08-12 爱康泰生治疗公司 用于递送核酸的改进的脂质纳米颗粒
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4058032A4 (de) 2019-12-19 2024-01-10 Entrada Therapeutics, Inc. Zusammensetzungen zur verabreichung von antisense-verbindungen
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
EP4121018A4 (de) 2020-03-17 2024-07-03 Genevant Sciences Gmbh Kationische lipide zur lipidnanopartikelabgabe von therapeutika an hepatische stellatzellen
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
SI4126066T1 (sl) 2020-03-27 2026-04-30 Avidity Biosciences, Inc. Sestavki in postopki zdravljenja mišične distrofije
CA3176844A1 (en) 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
IL301253A (en) 2020-09-13 2023-05-01 Arcturus Therapeutics Inc As a mass of large RNA lipid nanoparticles
KR20230107296A (ko) 2020-11-09 2023-07-14 1이 테라퓨틱스 엘티디. 촉매 서열에 기반한 박테리아 감염을 치료 또는 예방하는 방법
IL303753A (en) 2020-12-18 2023-08-01 Yeda res & development co ltd Preparations for use in the treatment of CHD2 HAPLOINSUFFICIENCY and methods for detection
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
KR20230126725A (ko) 2020-12-28 2023-08-30 1이 테라퓨틱스 엘티디. 침묵화를 위한 P21 mRNA 표적 부위
KR20230133859A (ko) 2020-12-28 2023-09-19 1이 테라퓨틱스 엘티디. p21 mRNA 표적화 DNA자임
EP4314016A1 (de) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Zyklische zellen penetrierende peptide
CN117242173A (zh) 2021-05-03 2023-12-15 安斯泰来再生医药协会 产生成熟角膜内皮细胞的方法
EP4337263A1 (de) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Zusammensetzungen und verfahren zur modulation der interferon-regulatorischen faktor-5 (irf-5)-aktivität
AU2022271873A1 (en) 2021-05-10 2024-01-04 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
WO2022240760A2 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
US20240218351A1 (en) 2021-06-14 2024-07-04 2Seventy Bio, Inc. Single stranded rna purification methods
AU2022298774A1 (en) 2021-06-23 2023-12-14 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
US12465647B2 (en) 2021-08-05 2025-11-11 Sanegene Bio Usa Inc. 1′-alkyl modified ribose derivatives and methods of use
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
EP4405368A1 (de) 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl oder 3'-alkyl modifizierte ribose-derivate zur verwendung in der in-vivo-verabreichung von oligonukleotiden
EP4413015A1 (de) 2021-10-05 2024-08-14 Sanegene Bio USA Inc. Polyhydroxylierte cyclopentanderivate und anwendungsverfahren
CN118900701A (zh) 2022-01-31 2024-11-05 盖纳万科学有限公司 聚(烷基噁唑啉)-脂质缀合物及含有聚(烷基噁唑啉)-脂质缀合物的脂质颗粒
KR20240146674A (ko) 2022-01-31 2024-10-08 제네반트 사이언시즈 게엠베하 지질 나노입자를 위한 이온화 가능한 양이온성 지질
US20230346819A1 (en) 2022-02-22 2023-11-02 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
KR20230159804A (ko) 2022-05-13 2023-11-22 서울대학교산학협력단 DICER에 의한 dsRNA 가공의 서열 결정 인자
EP4555088A1 (de) 2022-07-11 2025-05-21 Sanegene Bio USA Inc. Optimierte 2'-modifizierte ribosederivate und verfahren zur verwendung
KR20250052378A (ko) 2022-07-21 2025-04-18 안티바 바이오사이언시즈, 인크. Hpv 감염 및 hpv-유도 신생물의 치료를 위한 조성물 및 투여 형태
MX2025013094A (es) 2023-05-03 2026-02-03 Sanegene Bio Usa Inc Conjugados basados en lipidos para la administracion sistemica en el sistema nervioso central, el sistema nervioso periferico y ocular
EP4735602A2 (de) 2023-06-27 2026-05-06 Atrium Therapeutics, Inc. Zusammensetzungen und verfahren zur verwendung eines auf prkag2 abzielenden antikörper-oligonukleotid-konjugat-abstrahs
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
WO2025052278A1 (en) 2023-09-05 2025-03-13 Genevant Sciences Gmbh Pyrrolidine based cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
WO2025153530A1 (en) 2024-01-16 2025-07-24 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4987071A (en) * 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5149796A (en) * 1989-08-31 1992-09-22 City Of Hope Chimeric DNA-RNA catalytic sequences
AU637800B2 (en) * 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
JPH05509224A (ja) * 1990-06-19 1993-12-22 コモンウェルス　サイエンティフィク　アンド　インダストリアル　リサーチ　オーガナイゼイション エンドヌクレアーゼ
ES2061416T3 (es) * 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
DE4216134A1 (de) * 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US6204027B1 (en) * 1992-02-26 2001-03-20 University Of Massachusetts Worcester Ribozymes having 2′-O substituted nucleotides in the flanking sequences

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EP0625192A1 (de) 1994-11-23
EP1162263A1 (de) 2001-12-12
MX9300499A (es) 1994-07-29
US6140491A (en) 2000-10-31
AU3600193A (en) 1993-09-01
US5652094A (en) 1997-07-29
AU680405B2 (en) 1997-07-31
DE69331946T2 (de) 2003-01-02
EP0625192B1 (de) 2002-05-22
JPH07503613A (ja) 1995-04-20
CA2129119A1 (en) 1993-08-05
EP1219709A1 (de) 2002-07-03
DE69331946D1 (de) 2002-06-27
WO1993015187A1 (en) 1993-08-05
US6713456B1 (en) 2004-03-30
US20020137718A1 (en) 2002-09-26
US7074601B2 (en) 2006-07-11

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