CA2936534C - Methods and compositions for rnai mediated inhibition of gene expression in mammals - Google Patents

Methods and compositions for rnai mediated inhibition of gene expression in mammals Download PDF

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Publication number
CA2936534C
CA2936534C CA2936534A CA2936534A CA2936534C CA 2936534 C CA2936534 C CA 2936534C CA 2936534 A CA2936534 A CA 2936534A CA 2936534 A CA2936534 A CA 2936534A CA 2936534 C CA2936534 C CA 2936534C
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CA
Canada
Prior art keywords
rna
sirna
rnai agent
expression
gene
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
CA2936534A
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English (en)
French (fr)
Other versions
CA2936534A1 (en
Inventor
Mark Kay
Anton Mccaffrey
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Leland Stanford Junior University
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Leland Stanford Junior University
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Expired - Lifetime legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Environmental Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (AREA)
  • Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Rheumatology (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
CA2936534A 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals Expired - Lifetime CA2936534C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US30741101P 2001-07-23 2001-07-23
US60/307,441 2001-07-24
US36066402P 2002-02-27 2002-02-27
US60/360,664 2002-02-27
CA2454183A CA2454183C (en) 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2454183A Division CA2454183C (en) 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals

Publications (2)

Publication Number Publication Date
CA2936534A1 CA2936534A1 (en) 2003-02-06
CA2936534C true CA2936534C (en) 2021-01-26

Family

ID=26975733

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2936534A Expired - Lifetime CA2936534C (en) 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals
CA2454183A Expired - Lifetime CA2454183C (en) 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals

Family Applications After (1)

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CA2454183A Expired - Lifetime CA2454183C (en) 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals

Country Status (9)

Country Link
US (3) US20030153519A1 (OSRAM)
EP (2) EP2280070B1 (OSRAM)
JP (4) JP2005508306A (OSRAM)
AT (1) ATE556720T1 (OSRAM)
CA (2) CA2936534C (OSRAM)
DK (2) DK2280070T3 (OSRAM)
ES (2) ES2546829T3 (OSRAM)
PT (1) PT2280070E (OSRAM)
WO (1) WO2003010180A1 (OSRAM)

Families Citing this family (76)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells
US20050222075A1 (en) * 1998-09-14 2005-10-06 Hans Herweijer Process for delivering nucleic acids to cardiac tissue
IL145778A0 (en) * 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
US20040138168A1 (en) * 1999-04-21 2004-07-15 Wyeth Methods and compositions for inhibiting the function of polynucleotide sequences
CA2403397A1 (en) 2000-03-16 2001-09-20 Genetica, Inc. Methods and compositions for rna interference
US8202846B2 (en) 2000-03-16 2012-06-19 Cold Spring Harbor Laboratory Methods and compositions for RNA interference
ATE556720T1 (de) 2001-07-23 2012-05-15 Univ Leland Stanford Junior Verfahren und zusammensetzungen zur rnai vermittelten inhibierung der genexpression in säugetieren
US10590418B2 (en) * 2001-07-23 2020-03-17 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for RNAi mediated inhibition of gene expression in mammals
US7101995B2 (en) * 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
ATE356223T1 (de) 2001-09-25 2007-03-15 Japan President Univ Tokyo Gen und protein mit bezug zu hepatozellulärem karzinom
EP2455467B1 (en) 2002-02-01 2016-10-05 Life Technologies Corporation Double-stranded oligonucleotides
EP1572902B1 (en) 2002-02-01 2014-06-11 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
EP1495120B1 (en) * 2002-04-18 2012-10-10 Acuity Pharmaceuticals, Inc Means and methods for the specific modulation of target genes in the eye
US20030228691A1 (en) * 2002-05-17 2003-12-11 Lewis David L. Processes for inhibiting gene expression using polynucleotides
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
EP1532248B1 (en) * 2002-07-26 2009-04-01 Novartis Vaccines and Diagnostics, Inc. Modified small interfering rna molecules and methods of use
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
EP2112229A3 (en) 2002-11-25 2009-12-02 Sequenom, Inc. Methods for identifying risk of breast cancer and treatments thereof
US7781415B2 (en) * 2003-02-07 2010-08-24 Roche Madison Inc. Process for delivering sirna to cardiac muscle tissue
CN1780912B (zh) * 2003-02-28 2011-06-15 肿瘤疗法科学股份有限公司 抑制细胞生长的组合物和方法
ES2439580T5 (en) * 2003-02-28 2025-01-23 Univ Johns Hopkins T cell regulation
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
NZ574973A (en) * 2003-06-06 2010-10-29 Senesco Technologies Inc Inhibition of apoptosis-specific eIF-5A ("eIF-5A1") with antisense oligonucleotides and siRNAs as anti-inflammatory therapeutics
US20050060771A1 (en) * 2003-09-11 2005-03-17 Farmer Andrew Alan siRNA encoding constructs and methods for using the same
TWI350373B (en) 2004-01-23 2011-10-11 Oncotherapy Science Inc Methods of detecting methyl transferase activity and methods of screening formethyl transferase activity modulators
EP1737956A2 (en) * 2004-03-01 2007-01-03 Massachusetts Institute of Technology Rnai-based therapeutics for allergic rhinitis and asthma
US20050233994A1 (en) * 2004-04-16 2005-10-20 Ajamete Kaykas Methods and vectors for expressing siRNA
WO2005118640A2 (en) * 2004-04-20 2005-12-15 University Of Utah Research Foundation A h+-gated ion channel
JP4595939B2 (ja) * 2004-06-22 2010-12-08 株式会社ニコン 質感を強調する画像処理装置、画像処理プログラム、電子カメラ、および画像処理方法
US20060063208A1 (en) 2004-08-02 2006-03-23 Woolf Clifford J DRG11-responsive (DRAGON) gene and uses thereof
EP1778841A2 (en) * 2004-08-11 2007-05-02 Receptor Biologix, Inc. Gastrin-specific interfering rna
JP2008512500A (ja) 2004-09-10 2008-04-24 ソマジェニックス インコーポレーティッド ウィルス遺伝子発現を効率的に阻害する低分子干渉ｒｎａおよびその使用方法
US8765704B1 (en) 2008-02-28 2014-07-01 Novartis Ag Modified small interfering RNA molecules and methods of use
EP1796732B1 (en) * 2004-10-01 2013-10-30 Novartis Vaccines and Diagnostics, Inc. Modified small interfering rna molecules and methods of use
AU2005311684A1 (en) 2004-12-02 2006-06-08 University Of Massachusetts Glucose-transport related genes, polypeptides, and methods of use thereof
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
BRPI0519690A2 (pt) * 2004-12-30 2009-03-03 Todd M Hauser composiÇÕes e mÉtodos para modular a expressço de genes usando oligonucleotÍdeos autoprotegidos
ES2386549T3 (es) * 2005-01-28 2012-08-22 Kyowa Hakko Kirin Co., Ltd. Composición para inhibir la expresión de un gen diana
CN101189346A (zh) 2005-02-28 2008-05-28 肿瘤疗法科学股份有限公司 乳腺癌相关基因znfn3a1
EP1714970A1 (en) * 2005-04-22 2006-10-25 Universität des Saarlandes Use of inhibitors of RNAse A-family enzymes for stabilizing oligonucleotides having RNA interfering activity
TW200741009A (en) 2005-07-01 2007-11-01 Oncotherapy Science Inc Methods of modulating SMYD3 for treatment of cancer
US7919583B2 (en) * 2005-08-08 2011-04-05 Discovery Genomics, Inc. Integration-site directed vector systems
US7723314B1 (en) 2005-10-28 2010-05-25 Transderm, Inc. Methods and compositions for treating pachyonychia congenita
EP1970078A4 (en) 2006-01-11 2010-11-17 Kyowa Hakko Kirin Co Ltd COMPOSITION INHIBITING THE EXPRESSION OF A TARGET GENE OF THE OCULAR GLOBE AND REMEDY FOR OCULAR GLOBE DISEASE
US9150882B2 (en) 2006-01-31 2015-10-06 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
EP1991008A4 (en) * 2006-02-27 2012-04-18 Nikon Corp IMAGE PROCESSING DEVICE FOR CORRECTING MASSIVE IMAGE PERCEPTION, IMAGE PROCESSING PROGRAM AND METHOD, AND ELECTRONIC CAMERA
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
US8153369B2 (en) 2006-06-05 2012-04-10 Cancer Care Ontario Assessment of risk for colorectal cancer
ES2390499T3 (es) * 2006-06-12 2012-11-13 Opko Pharmaceuticals, Llc Composiciones y métodos para la inhibición de la angiogénesis por sirna
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
WO2008106785A1 (en) 2007-03-05 2008-09-12 Cancer Care Ontario Assessment of risk for colorectal cancer
WO2008152816A1 (en) 2007-06-14 2008-12-18 Oncotherapy Science, Inc. Methods of identifying agents that modulate methylation of vegfr1 by smyd3
EP2170395A1 (en) * 2007-07-02 2010-04-07 Wyeth LLC Modulators of axl for use in treating bone disorders
WO2010008562A2 (en) 2008-07-16 2010-01-21 Recombinetics Methods and materials for producing transgenic animals
KR101630888B1 (ko) 2008-08-01 2016-06-15 교와 핫꼬 기린 가부시키가이샤 표적 유전자의 발현을 억제하는 조성물
MX2011005851A (es) 2008-12-04 2011-07-29 Opko Opthalmics Llc Composiciones y metodos para la inhibicion selectiva de isoformas vegf proangiogenicas.
ES2804764T3 (es) 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
US8871730B2 (en) 2009-07-13 2014-10-28 Somagenics Inc. Chemical modification of short small hairpin RNAs for inhibition of gene expression
EP2666856A4 (en) 2011-01-19 2015-01-14 Kyowa Hakko Kirin Co Ltd COMPOSITION TO INHIBIT TARGET EXPRESSION
CA2834788C (en) 2011-05-05 2020-11-17 Coordinated Program Development, Llc Cochleate compositions and methods of making and using same
EP2873732A4 (en) 2012-07-16 2016-03-23 Kyowa Hakko Kirin Co Ltd PHARMACEUTICAL RNAI COMPOSITION WITH SUPPRESSION OF KRAS GENE EXPRESSION
US9655977B2 (en) * 2012-08-31 2017-05-23 The General Hospital Corporation Biotin complexes for treatment and diagnosis of alzheimer's disease
CN104342453A (zh) 2013-08-06 2015-02-11 深圳先进技术研究院 含基因工程抗体基因表达盒的微环dna重组母质粒、含该表达盒的微环dna及应用
US9545417B2 (en) 2014-05-01 2017-01-17 The Johns Hopkins University Methods of inhibiting cancer stem cells with HMGA1 inhibitors
CN106794194A (zh) 2014-06-30 2017-05-31 中央研究院 用于癌症治疗的介白素‑17受体b（il‑17rb）及其配体il‑17b之拮抗剂
WO2016022947A1 (en) 2014-08-07 2016-02-11 The Johns Hopkins University Nanoparticle modification of human adipose-derived mesenchymal stem cells for treating brain cancer and other neurological diseases
WO2016025629A1 (en) 2014-08-12 2016-02-18 The Regents Of The University Of California Molecular composition for enhancing and rejuvenating maintenance and repair of mammalian tissues
EP3188746B1 (en) 2014-09-05 2024-06-19 The Johns Hopkins University Targeting capn9 activity as a therapeutic strategy for the treatment of myofibroblast differentiation and associated pathologies
NZ738184A (en) 2015-05-18 2019-09-27 Univ California Methods and compositions for treating aging-associated impairments
CA2995995A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
WO2018009417A1 (en) 2016-07-05 2018-01-11 Blade Therapeutics, Inc. Calpain modulators and therapeutic uses thereof
JP2020500207A (ja) 2016-09-28 2020-01-09 ブレード・セラピューティクス・インコーポレイテッド カルパインモジュレーター及びそれらの治療上の使用
US12031161B2 (en) 2020-10-09 2024-07-09 University Of Massachusetts Targeting Nrip1 to alleviate metabolic disease

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69034168T3 (de) 1989-03-21 2013-04-11 Vical, Inc. Expression von exogenen Polynukleotidsequenzen in Wirbeltieren
US5610050A (en) * 1990-04-20 1997-03-11 The General Hospital Corporation Methods of preventing viral replication
US6344321B1 (en) 1990-06-11 2002-02-05 Gilead Sciences, Inc. Nucleic acid ligands which bind to hepatocyte growth factor/scatter factor (HGF/SF) or its receptor c-met
CA2038839A1 (en) * 1991-03-22 1992-09-23 Jiang J. Liu Method for detection of viral rna
ES2040615B1 (es) * 1991-05-27 1994-05-16 Nebrda Fernando Javi Bartolome Procedimiento para el aislamiento y clonaje de cdnas derivados del virus c de la hepatitis.
US6030954A (en) * 1991-09-05 2000-02-29 University Of Connecticut Targeted delivery of poly- or oligonucleotides to cells
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
US6174868B1 (en) * 1992-09-10 2001-01-16 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis C virus-associated diseases
JP3615545B2 (ja) * 1993-02-01 2005-02-02 キアゲン・エヌ・ブイ 第四級アンモニウム塩界面活性剤及びそのｒｎａの単離剤
US5985847A (en) 1993-08-26 1999-11-16 The Regents Of The University Of California Devices for administration of naked polynucleotides which encode biologically active peptides
US5712384A (en) * 1994-01-05 1998-01-27 Gene Shears Pty Ltd. Ribozymes targeting retroviral packaging sequence expression constructs and recombinant retroviruses containing such constructs
EP0759979A4 (en) * 1994-05-10 1999-10-20 Gen Hospital Corp THE ANTISENSE INHIBITION OF HEPATITIS C VIRUS
US6107027A (en) * 1994-12-14 2000-08-22 University Of Washington Ribozymes for treating hepatitis C
US5922687A (en) * 1995-05-04 1999-07-13 Board Of Trustees Of The Leland Stanford Junior University Intracellular delivery of nucleic acids using pressure
US5981279A (en) * 1995-11-09 1999-11-09 Allegheny University Of The Health Sciences Compositions and methods to regulate calmodulin gene expression, and uses thereof for influencing cell growth and differentiation
JP4033502B2 (ja) 1996-02-08 2008-01-16 アンジェスＭｇ株式会社 リボザイム、リポソーム製剤及びその用途
WO1997036005A1 (en) * 1996-03-26 1997-10-02 Lynx Therapeutics, Inc. Oligonucleotide treatments and compositions for human melanoma
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
WO1998044953A1 (en) 1997-04-04 1998-10-15 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Methods for the modulation of the growth of collateral arteries and/or other arteries from preexisting arteriolar connections
US5948902A (en) 1997-11-20 1999-09-07 South Alabama Medical Science Foundation Antisense oligonucleotides to human serine/threonine protein phosphatase genes
US5957978A (en) 1997-12-22 1999-09-28 Hansa Medical Products, Inc. Valved fenestrated tracheotomy tube
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
NZ506648A (en) 1998-03-20 2003-08-29 Benitec Australia Ltd Control of gene expression through introduction of synthetic tandem repeats to reduce translation of mRNA
AUPP249298A0 (en) * 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
IL145778A0 (en) * 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
GB9925459D0 (en) * 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
ES2269199T3 (es) 1999-11-05 2007-04-01 Mirus Bio Corporation Suministro intravascular de acido nucleico.
GB9927444D0 (en) * 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE10100586C1 (de) * 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
CA2403397A1 (en) 2000-03-16 2001-09-20 Genetica, Inc. Methods and compositions for rna interference
US20030084471A1 (en) 2000-03-16 2003-05-01 David Beach Methods and compositions for RNA interference
BR0109269A (pt) 2000-03-17 2002-12-17 Benitec Australia Ltd Construto genético e seu uso, célula de animal vertebrado, métodos de alteração do fenótipo de uma célula de animal vertebrado e de terapia genética em animal vertebrado, animal e animal murino geneticamente modificados
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
US20020132788A1 (en) * 2000-11-06 2002-09-19 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
CZ302719B6 (cs) * 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
DE10100588A1 (de) 2001-01-09 2002-07-18 Ribopharma Ag Verfahren zur Hemmung der Expression eines Zielgens
EP1390472A4 (en) * 2001-05-29 2004-11-17 Sirna Therapeutics Inc NUCLEIC ACID TREATMENT OF DISEASES OR SIDES RELATED TO RAS, HER2 AND HIV LEVELS
US7000102B2 (en) 2001-06-29 2006-02-14 Intel Corporation Platform and method for supporting hibernate operations
EP1412371B1 (en) 2001-07-12 2016-02-24 University of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
ATE556720T1 (de) 2001-07-23 2012-05-15 Univ Leland Stanford Junior Verfahren und zusammensetzungen zur rnai vermittelten inhibierung der genexpression in säugetieren
JP2005517427A (ja) 2002-02-20 2005-06-16 サーナ・セラピューティクス・インコーポレイテッド 短干渉核酸（ｓｉＮＡ）を用いるＣ型肝炎ウイルス（ＨＣＶ）遺伝子発現のＲＮＡ干渉媒介性阻害

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