CA2624673A1 - Allele-specific rna interference - Google Patents

Allele-specific rna interference Download PDF

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Publication number
CA2624673A1
CA2624673A1 CA002624673A CA2624673A CA2624673A1 CA 2624673 A1 CA2624673 A1 CA 2624673A1 CA 002624673 A CA002624673 A CA 002624673A CA 2624673 A CA2624673 A CA 2624673A CA 2624673 A1 CA2624673 A1 CA 2624673A1
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Prior art keywords
shrna
sirna
base pair
strand portion
target
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Abandoned
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CA002624673A
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English (en)
French (fr)
Inventor
Zuoshang Xu
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University of Massachusetts Amherst
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Individual
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Publication of CA2624673A1 publication Critical patent/CA2624673A1/en
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    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA002624673A 2005-09-30 2006-10-02 Allele-specific rna interference Abandoned CA2624673A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11/241,873 US7892793B2 (en) 2002-11-04 2005-09-30 Allele-specific RNA interference
US11/241,873 2005-09-30
PCT/US2006/038704 WO2007044362A2 (en) 2005-09-30 2006-10-02 Allele-specific rna interference

Publications (1)

Publication Number Publication Date
CA2624673A1 true CA2624673A1 (en) 2007-04-19

Family

ID=37943327

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002624673A Abandoned CA2624673A1 (en) 2005-09-30 2006-10-02 Allele-specific rna interference

Country Status (6)

Country Link
US (2) US7892793B2 (enExample)
EP (1) EP1940862A4 (enExample)
JP (1) JP2009509548A (enExample)
AU (1) AU2006302534A1 (enExample)
CA (1) CA2624673A1 (enExample)
WO (1) WO2007044362A2 (enExample)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7892793B2 (en) * 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
CA2527958C (en) * 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
CA2528012C (en) * 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2005027980A1 (en) * 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
EP2239328A3 (en) 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
WO2007030167A1 (en) * 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
WO2008016988A1 (en) 2006-08-01 2008-02-07 Gen-Probe Incorporated Methods of nonspecific target capture of nucleic acids
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー.フェー. Dnaリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
WO2008147839A1 (en) 2007-05-23 2008-12-04 Dharmacon, Inc. Micro-rna scaffolds and non-naturally occurring micro-rnas
JP2010533170A (ja) 2007-07-12 2010-10-21 プロセンサ テクノロジーズ ビー.ブイ. 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2009062169A2 (en) 2007-11-09 2009-05-14 The Board Of Regents Of The University Of Texas System Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair
JP2011510678A (ja) 2008-02-08 2011-04-07 プロセンサ ホールディング ビーブイ Dna反復不安定性関連遺伝性障害を治療するための方法及び手段
EP3643782A1 (en) * 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010033225A2 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
KR20110110776A (ko) * 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
ES2609655T3 (es) 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP
EP2427554B1 (en) * 2009-05-08 2016-11-16 CuRNA, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
WO2011119852A1 (en) 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
US20110306653A1 (en) 2010-05-14 2011-12-15 Tagcyx Biotechnologies Stabilization method of functional nucleic acid
GB201105137D0 (en) * 2011-03-28 2011-05-11 Isis Innovation Therapeutic molecules for use in the suppression of Parkinson's disease
WO2013086041A1 (en) * 2011-12-05 2013-06-13 The Trustees Of Columbia University In The City Of New York Transcriptome wiring analysis in parkinson's disease and uses thereof
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
PT2841578T (pt) 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
US9163235B2 (en) 2012-06-21 2015-10-20 MiRagen Therapeutics, Inc. Inhibitors of the miR-15 family of micro-RNAs
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのrAAVベースの組成物および方法
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
US11001845B2 (en) 2015-07-06 2021-05-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)
JP2019515654A (ja) 2016-03-16 2019-06-13 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 肥満及び/又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物
BR112019007719A2 (pt) * 2016-10-19 2019-07-09 Devgen Nv ácidos nucleicos compreendendo forquilhas imperfeitas
US10947540B2 (en) * 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases
US11613754B2 (en) * 2017-02-20 2023-03-28 Northwestern University Toxic RNAi active seed sequences for killing cancer cells
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018264996A1 (en) 2017-05-09 2019-12-05 University Of Massachusetts Methods of treating Amyotrophic Lateral Sclerosis (ALS)
CN118006607A (zh) 2017-09-22 2024-05-10 马萨诸塞大学 Sod1双表达载体及其用途
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
US11946047B2 (en) * 2018-10-23 2024-04-02 Texas Tech University System Treatment strategies against anthrax by interfering with critical host factors
WO2020112736A1 (en) * 2018-11-30 2020-06-04 Dow Agrosciences Llc Extension sequences for rna inhibitory molecules
WO2021174168A1 (en) * 2020-02-28 2021-09-02 Blueallele, Llc Methods for treating gain-of-function disorders combining gene editing and gene therapy
WO2023191957A1 (en) * 2022-03-30 2023-10-05 Mirimus, Inc. Compositions and methods of generating novel amirna
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5843641A (en) * 1993-02-26 1998-12-01 Massachusetts Institute Of Technology Methods for the daignosis, of familial amyotrophic lateral sclerosis
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
WO2003035869A1 (de) 2001-10-26 2003-05-01 Ribopharma Ag Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
BR0211111A (pt) 2001-07-12 2004-06-22 Univ Massachusetts Molécula de ácido nucleico isolada, vetor, célula hospedeira, transgene, precursor de rna engenheirado, animal transgênico não humano, e, método de induzir a interferência de ácido ribonucleico de um gene alvo em uma célula
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
EP2428570B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
JP2005527198A (ja) 2002-02-14 2005-09-15 シティ・オブ・ホープ 哺乳動物細胞において、干渉rna分子を産生する方法、および該干渉rna分子の療法的使用
CA2489174C (en) 2002-07-10 2013-02-05 Thomas Tuschl Rna-interference by single-stranded rna molecules
ES2389024T3 (es) 2002-08-05 2012-10-22 Silence Therapeutics Aktiengesellschaft Moléculas de RNA interferentes de extremos romos
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
AU2003261449A1 (en) 2002-08-07 2004-02-25 Compositions for rna interference and methods of use thereof
WO2004029212A2 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
AU2003290586B2 (en) 2002-11-04 2009-07-02 University Of Massachusetts Allele-specific RNA interference
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2004046324A2 (en) 2002-11-15 2004-06-03 University Of Massachusetts Allele-targeted rna interference
US20040248299A1 (en) 2002-12-27 2004-12-09 Sumedha Jayasena RNA interference
EP1583832B1 (en) 2003-01-17 2010-12-01 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Inducible small interfering rna (sirna) expression constructs for targeted gene silencing
US20070104688A1 (en) 2003-02-13 2007-05-10 City Of Hope Small interfering RNA mediated transcriptional gene silencing in mammalian cells
CA2528012C (en) 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
CA2527958C (en) 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
US8685946B2 (en) 2003-11-26 2014-04-01 Universiy of Massachusetts Sequence-specific inhibition of small RNA function
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
US20050227940A1 (en) 2004-01-23 2005-10-13 City Of Hope Amplifying interfering RNA (RNAi) expression and effects
WO2005078096A2 (en) 2004-02-09 2005-08-25 University Of Massachusetts Dual functional oligonucleotides for use in repressing mutant gene expression
US20050182005A1 (en) 2004-02-13 2005-08-18 Tuschl Thomas H. Anti-microRNA oligonucleotide molecules
US20060069050A1 (en) 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
WO2005079532A2 (en) 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for enhancing risc activity in vitro and in vivo
CA2559955C (en) 2004-03-15 2016-02-16 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20060178334A1 (en) 2005-02-04 2006-08-10 City Of Hope Double-stranded and single-stranded RNA molecules with 5 ' triphosphates and their use for inducing interferon
US20070207874A1 (en) * 2006-03-03 2007-09-06 Klein Bernard E Golf ball with optical feedback for improving putting stroke

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EP1940862A4 (en) 2009-01-07
EP1940862A2 (en) 2008-07-09
JP2009509548A (ja) 2009-03-12
US7892793B2 (en) 2011-02-22
US20060128650A1 (en) 2006-06-15
WO2007044362A2 (en) 2007-04-19
US20110160286A1 (en) 2011-06-30

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