JP2010500025A - 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列 - Google Patents

霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列 Download PDF

Info

Publication number
JP2010500025A
JP2010500025A JP2009523833A JP2009523833A JP2010500025A JP 2010500025 A JP2010500025 A JP 2010500025A JP 2009523833 A JP2009523833 A JP 2009523833A JP 2009523833 A JP2009523833 A JP 2009523833A JP 2010500025 A JP2010500025 A JP 2010500025A
Authority
JP
Japan
Prior art keywords
nucleic acid
strand
seq
expression
vector
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.)
Pending
Application number
JP2009523833A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010500025A5 (enExample
Inventor
ケンメラー，ウィリアム・エフ
ケイトー，マイケル・ディー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Medtronic Inc
Original Assignee
Medtronic Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Medtronic Inc filed Critical Medtronic Inc
Publication of JP2010500025A publication Critical patent/JP2010500025A/ja
Publication of JP2010500025A5 publication Critical patent/JP2010500025A5/ja
Pending legal-status Critical Current

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
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2009523833A 2006-08-09 2007-08-08 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列 Pending JP2010500025A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11/501,634 US7902352B2 (en) 2005-05-06 2006-08-09 Isolated nucleic acid duplex for reducing huntington gene expression
PCT/US2007/017638 WO2008021136A2 (en) 2006-08-09 2007-08-08 Methods and sequences to suppress primate huntington gene expression in vivo

Publications (2)

Publication Number Publication Date
JP2010500025A true JP2010500025A (ja) 2010-01-07
JP2010500025A5 JP2010500025A5 (enExample) 2011-10-20

Family

ID=39082580

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2009523833A Pending JP2010500025A (ja) 2006-08-09 2007-08-08 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列

Country Status (5)

Country Link
US (1) US7902352B2 (enExample)
EP (1) EP2056881A4 (enExample)
JP (1) JP2010500025A (enExample)
CN (1) CN101557831A (enExample)
WO (1) WO2008021136A2 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018506304A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
US11603529B2 (en) 2017-09-22 2023-03-14 Genzyme Corporation Variant RNAi

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
ES2485848T3 (es) * 2003-09-12 2014-08-14 University Of Massachusetts ARN de interferencia para el tratamiento de trastornos relacionados con la ganancia de función
US20060257912A1 (en) * 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
WO2007022470A2 (en) * 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
US8227592B2 (en) * 2006-11-29 2012-07-24 University Of Iowa Research Foundation Alternative export pathways for vector expressed RNA interference
WO2008134646A2 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
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
US9738694B2 (en) 2008-06-30 2017-08-22 Cho-A Pharm. Co., Ltd. Gene of porcine alpha-s1 casein, a promoter of the same and use thereof
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
JP6663859B2 (ja) 2014-05-20 2020-03-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション ハンチントン病の治療化合物
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
MX2023011466A (es) 2021-03-29 2024-02-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas.

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004013280A2 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation ALLELE-SPECIFIC siRNA-MEDIATED GENE SILENCING
WO2005116212A2 (en) * 2004-05-25 2005-12-08 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of sirna
US20060009408A1 (en) * 2002-08-05 2006-01-12 University Of Iowa Research Foundation, A Iowa Corporation siRNA-Mediated gene silencing with viral vectors
JP2006515864A (ja) * 2002-11-26 2006-06-08 メドトロニック・インコーポレーテッド ｓｉＲＮＡの頭蓋内送達を通した神経変性疾患の治療

Family Cites Families (119)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4965188A (en) * 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683195A (en) * 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) * 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
GB2181691B (en) * 1985-10-21 1990-05-23 Porvair Ltd Gloves
US4800159A (en) * 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5702716A (en) * 1988-10-03 1997-12-30 Atrix Laboratories, Inc. Polymeric compositions useful as controlled release implants
US5800390A (en) * 1991-05-24 1998-09-01 Sumitomo Pharmaceuticals Company, Limited Equipment for intracerebral administration of preparations
US5236908A (en) * 1991-06-07 1993-08-17 Gensia Pharmaceuticals, Inc. Methods of treating injury to the central nervous system
US5354326A (en) * 1993-01-27 1994-10-11 Medtronic, Inc. Screening cable connector for interface to implanted lead
JP4098355B2 (ja) * 1993-08-12 2008-06-11 ニューロテック ユーエスエー， インコーポレイテッド 遺伝的に改変された細胞を含む生体適合性免疫隔離カプセル
WO1995005864A1 (en) * 1993-08-27 1995-03-02 Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Convection-enhanced drug delivery
US5849995A (en) * 1993-09-27 1998-12-15 The University Of British Columbia Mouse model for Huntington's Disease and related DNA sequences
US5624803A (en) * 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
EP0734438A4 (en) * 1993-12-17 1998-12-23 Spinal Cord Society METHOD FOR INDUCING DNA SYNTHESIS IN NEURONES
WO1995028493A1 (en) * 1994-04-13 1995-10-26 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US6294202B1 (en) * 1994-10-06 2001-09-25 Genzyme Corporation Compositions containing polyanionic polysaccharides and hydrophobic bioabsorbable polymers
US5534350A (en) * 1994-12-28 1996-07-09 Liou; Derlin Powerfree glove and its making method
US7069634B1 (en) 1995-04-28 2006-07-04 Medtronic, Inc. Method for manufacturing a catheter
WO1996033761A1 (en) * 1995-04-28 1996-10-31 Medtronic, Inc. Intraparenchymal infusion catheter system
US5840059A (en) * 1995-06-07 1998-11-24 Cardiogenesis Corporation Therapeutic and diagnostic agent delivery
US5942455A (en) * 1995-11-14 1999-08-24 Drexel University Synthesis of 312 phases and composites thereof
JPH09268067A (ja) * 1996-03-29 1997-10-14 Asahi Glass Co Ltd 炭化ケイ素部材の製造方法
WO2000062828A1 (en) * 1996-04-30 2000-10-26 Medtronic, Inc. Autologous fibrin sealant and method for making the same
US7189222B2 (en) * 1996-04-30 2007-03-13 Medtronic, Inc. Therapeutic method of treatment of alzheimer's disease
US5976109A (en) * 1996-04-30 1999-11-02 Medtronic, Inc. Apparatus for drug infusion implanted within a living body
US5735814A (en) * 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
WO1998012311A2 (en) * 1996-09-11 1998-03-26 The General Hospital Corporation Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
US5882561A (en) * 1996-11-22 1999-03-16 Drexel University Process for making a dense ceramic workpiece
GB9701684D0 (en) * 1997-01-28 1997-03-19 Smithkline Beecham Plc Novel compounds
US5968059A (en) * 1997-03-06 1999-10-19 Scimed Life Systems, Inc. Transmyocardial revascularization catheter and method
GB9706463D0 (en) * 1997-03-27 1997-05-14 Medical Res Council A model of inflamation in the central nervous system for use in the study of disease
JP2000514094A (ja) * 1997-04-17 2000-10-24 レオネ，パオラ 脳への遺伝子治療のための送達システム
US5782892A (en) * 1997-04-25 1998-07-21 Medtronic, Inc. Medical lead adaptor for external medical device
US5931861A (en) * 1997-04-25 1999-08-03 Medtronic, Inc. Medical lead adaptor having rotatable locking clip mechanism
US6042579A (en) * 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US6110459A (en) * 1997-05-28 2000-08-29 Mickle; Donald A. G. Transplants for myocardial scars and methods and cellular preparations
US20050282198A1 (en) 1997-05-29 2005-12-22 Interleukin Genetics, Inc. Diagnostics and therapeutics for diseases associated with an IL-1 inflammatory haplotype
US6187906B1 (en) * 1997-08-11 2001-02-13 Aukland Uniservices Limited Methods to improve neural outcome
US6231969B1 (en) * 1997-08-11 2001-05-15 Drexel University Corrosion, oxidation and/or wear-resistant coatings
US6376471B1 (en) * 1997-10-10 2002-04-23 Johns Hopkins University Gene delivery compositions and methods
US6151525A (en) * 1997-11-07 2000-11-21 Medtronic, Inc. Method and system for myocardial identifier repair
DE19938960A1 (de) 1998-03-10 2001-02-22 Bisping Hans Juergen Testkabelanordnung
US6436392B1 (en) * 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
PT1080202E (pt) * 1998-05-27 2006-05-31 Avigen Inc Distribuicao de vectores aav codificando aadc intensificada por conveccao
US6313268B1 (en) * 1998-10-16 2001-11-06 Vivian Y. H. Hook Secretases related to Alzheimer's dementia
US6245884B1 (en) * 1998-10-16 2001-06-12 Vivian Y. H. Hook Secretases related to alzheimer's dementia
US6319905B1 (en) * 1998-12-29 2001-11-20 Cell Genesys, Inc. Method of controlling L-Dopa production and of treating dopamine deficiency
US6835194B2 (en) * 1999-03-18 2004-12-28 Durect Corporation Implantable devices and methods for treatment of pain by delivery of fentanyl and fentanyl congeners
US6291243B1 (en) * 1999-04-28 2001-09-18 The Board Of Trustees Of The Leland Stanford Jr. University P element derived vector and methods for its use
GB9928248D0 (en) * 1999-12-01 2000-01-26 Gill Steven S An implantable guide tube for neurosurgery
US6310048B1 (en) * 1999-12-09 2001-10-30 St. Louis University Antisense modulation of amyloid beta protein expression
US6461989B1 (en) * 1999-12-22 2002-10-08 Drexel University Process for forming 312 phase materials and process for sintering the same
US20030092003A1 (en) * 1999-12-29 2003-05-15 Ribozyme Pharmaceuticals, Inc. Method and reagent for the treatment of Alzheimer's disease
US20050032733A1 (en) * 2001-05-18 2005-02-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
US20070026394A1 (en) * 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
CN1406140A (zh) * 2000-02-28 2003-03-26 吉倪塞思公司 纳米胶囊包封系统与方法
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6551290B1 (en) * 2000-03-31 2003-04-22 Medtronic, Inc. Catheter for target specific drug delivery
US6945969B1 (en) * 2000-03-31 2005-09-20 Medtronic, Inc. Catheter for target specific drug delivery
US6372250B1 (en) * 2000-04-25 2002-04-16 The Regents Of The University Of California Non-invasive gene targeting to the brain
US20020042388A1 (en) * 2001-05-01 2002-04-11 Cooper Mark J. Lyophilizable and enhanced compacted nucleic acids
US6866859B2 (en) * 2000-08-30 2005-03-15 Biocoat Incorporated Bi-laminar, hyaluronan coatings with silver-based anti-microbial properties
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US20050209179A1 (en) * 2000-08-30 2005-09-22 Sirna Therapeutics, Inc. RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
US6659995B1 (en) 2000-11-17 2003-12-09 Syde A. Taheri Autologous myocyte micro granual retrieval and implantation (AMMGRI)
CA2327208A1 (en) * 2000-11-30 2002-05-30 The Government Of The United States Of America Methods of increasing distribution of therapeutic agents
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
US7182944B2 (en) 2001-04-25 2007-02-27 The United States Of America As Represented By The Department Of Health And Human Services Methods of increasing distribution of nucleic acids
WO2002088101A2 (en) * 2001-04-27 2002-11-07 Vertex Pharmaceuticals Incorporated Inhibitors of bace
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20050191638A1 (en) * 2002-02-20 2005-09-01 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
CN100424182C (zh) * 2001-06-15 2008-10-08 白介素遗传公司 衰老相关疾病早期发作的检测与治疗方法
US20030109476A1 (en) * 2001-08-07 2003-06-12 Kmiec Eric B. Compositions and methods for the prevention and treatment of Huntington's disease
US6944497B2 (en) * 2001-10-31 2005-09-13 Medtronic, Inc. System and method of treating stuttering by neuromodulation
US8274559B2 (en) * 2001-11-09 2012-09-25 Karl Storz Imaging, Inc. Replaceable hardware component of a camera control unit for video systems
US20030120282A1 (en) * 2001-12-24 2003-06-26 Scouten Charles W. Stereotaxic manipulator with retrofitted linear scales and digital display device
US7294504B1 (en) * 2001-12-27 2007-11-13 Allele Biotechnology & Pharmaceuticals, Inc. Methods and compositions for DNA mediated gene silencing
US7270653B2 (en) * 2002-02-20 2007-09-18 Abbott Research Group Methods of treating abnormal biological conditions using metal oxides
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20030224512A1 (en) 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of beta-site APP-cleaving enzyme expression
US20040023855A1 (en) * 2002-04-08 2004-02-05 John Constance M. Biologic modulations with nanoparticles
US20040018520A1 (en) * 2002-04-22 2004-01-29 James Thompson Trans-splicing enzymatic nucleic acid mediated biopharmaceutical and protein
US7008403B1 (en) * 2002-07-19 2006-03-07 Cognitive Ventures Corporation Infusion pump and method for use
JP3968365B2 (ja) 2002-07-25 2007-08-29 日世株式会社 ロールコーン製造装置
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US8090542B2 (en) * 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
US20040265849A1 (en) 2002-11-22 2004-12-30 Applera Corporation Genetic polymorphisms associated with Alzheimer's disease, methods of detection and uses thereof
US20050048641A1 (en) * 2002-11-26 2005-03-03 Medtronic, Inc. System and method for delivering polynucleotides to the central nervous system
JP2004232811A (ja) 2003-01-31 2004-08-19 Hitachi Unisia Automotive Ltd 補機駆動用電磁クラッチ
US8946151B2 (en) * 2003-02-24 2015-02-03 Northern Bristol N.H.S. Trust Frenchay Hospital Method of treating Parkinson's disease in humans by convection-enhanced infusion of glial cell-line derived neurotrophic factor to the putamen
US8512290B2 (en) * 2003-03-20 2013-08-20 Boston Scientific Scimed, Inc. Devices and methods for delivering therapeutic or diagnostic agents
US20040186528A1 (en) 2003-03-20 2004-09-23 Medtronic, Inc. Subcutaneous implantable medical devices with anti-microbial agents for chronic release
US20040198640A1 (en) 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
US7917228B2 (en) 2003-05-13 2011-03-29 Medtronic, Inc. Medical lead adaptor assembly
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
US20060014165A1 (en) * 2003-07-14 2006-01-19 Decode Genetics Ehf. Methods of diagnosis and treatment for asthma and other respiratory diseases based on haplotype association
ES2485848T3 (es) 2003-09-12 2014-08-14 University Of Massachusetts ARN de interferencia para el tratamiento de trastornos relacionados con la ganancia de función
WO2005065242A2 (en) * 2003-12-29 2005-07-21 Am Biosolutions Method of treating cancer using platelet releasate
WO2005070104A2 (en) * 2004-01-09 2005-08-04 The University Of Tennessee Research Foundation REAL-TIME POLYMERASE CHAIN REACTION-BASED GENOTYPING ASSAY FOR β2-ADRENERGIC RECEPTOR SINGLE NUCLEODITE POLYMORPHISM
US20050202075A1 (en) * 2004-03-12 2005-09-15 Pardridge William M. Delivery of genes encoding short hairpin RNA using receptor-specific nanocontainers
WO2005096781A2 (en) 2004-04-06 2005-10-20 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using rna interference
EP1735443A2 (en) 2004-04-14 2006-12-27 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
AU2005248147A1 (en) * 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
WO2006022639A1 (en) 2004-07-21 2006-03-02 Applera Corporation Genetic polymorphisms associated with alzheimer's disease, methods of detection and uses thereof
WO2006047250A2 (en) * 2004-10-22 2006-05-04 Neurologix, Inc. Use of apoptosis inhibiting compounds in degenerative neurological disorders
NL1028134C2 (nl) * 2005-01-27 2006-07-31 Sara Lee De Nv Werkwijze voor het bereiden van een voor consumptie geschikte drank uit ten minste twee op te lossen en/of te extraheren ingredienten en een hoeveelheid vloeistof.
TW200635542A (en) * 2005-04-01 2006-10-16 Dharma Drum Mountain Method of establishing and using commemorative material
US20060257912A1 (en) * 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US20080280843A1 (en) 2006-05-24 2008-11-13 Van Bilsen Paul Methods and kits for linking polymorphic sequences to expanded repeat mutations
GB0520235D0 (en) 2005-10-05 2005-11-16 Astrazeneca Ab Method
CN101365801B (zh) * 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
PE20071241A1 (es) 2006-01-17 2008-01-14 Schering Corp Compuestos derivados de hidantoina para el tratamiento de trastornos inflamatorios
US20080039415A1 (en) 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
CN200970210Y (zh) 2006-10-12 2007-11-07 王会才 拖把清洗脱水机

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004013280A2 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation ALLELE-SPECIFIC siRNA-MEDIATED GENE SILENCING
US20060009408A1 (en) * 2002-08-05 2006-01-12 University Of Iowa Research Foundation, A Iowa Corporation siRNA-Mediated gene silencing with viral vectors
JP2006515864A (ja) * 2002-11-26 2006-06-08 メドトロニック・インコーポレーテッド ｓｉＲＮＡの頭蓋内送達を通した神経変性疾患の治療
WO2005116212A2 (en) * 2004-05-25 2005-12-08 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of sirna

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
JPN6012039014; Ｎｅｕｒｏｓｃｉｅｎｃｅ　Ｒｅｓｅａｒｃｈ ｖｏｌ．５３, 2005, ｐｐ．２４１-２４９ *
JPN6012039017; ＰＮＡＳ ｖｏｌ．１０２　ｎｏ．１６, 2005, ｐｐ．５８２０-５８２５ *
JPN6013007326; Ｄａｔａｂａｓｅ　ＧｅｎＢａｎｋ , 20060806, ＮＭ＿００２１１１ *
JPN6013007327; Ｄａｔａｂａｓｅ　ＧｅｎＢａｎｋ , 20001003, ＡＢ０１６７９３ *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018506304A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
US10760079B2 (en) 2015-02-10 2020-09-01 Genzyme Corporation Variant RNAi
US11781137B2 (en) 2015-02-10 2023-10-10 Genzyme Corporation Variant RNAi
US11603529B2 (en) 2017-09-22 2023-03-14 Genzyme Corporation Variant RNAi
US12331296B2 (en) 2017-09-22 2025-06-17 Genzyme Corporation Variant RNAi

Also Published As

Publication number Publication date
EP2056881A2 (en) 2009-05-13
EP2056881A4 (en) 2010-10-20
WO2008021136A2 (en) 2008-02-21
WO2008021136B1 (en) 2008-08-07
US7902352B2 (en) 2011-03-08
WO2008021136A3 (en) 2008-07-03
CN101557831A (zh) 2009-10-14
US20100325746A9 (en) 2010-12-23
US20070261126A1 (en) 2007-11-08

Similar Documents

Publication Publication Date Title
JP2010500025A (ja) 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列
US8258112B2 (en) Methods and sequences to suppress primate huntington gene Expression
Keiser et al. Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
Borel et al. Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques
Xu et al. In vivo gene knockdown in rat dorsal root ganglia mediated by self-complementary adeno-associated virus serotype 5 following intrathecal delivery
JP2017506228A (ja) セロトニン放出ホルモン、アドレナリン放出ホルモン、ノルアドレナリン放出ホルモン、グルタミン酸放出ホルモンおよび副腎皮質刺激ホルモン放出ホルモン関連の医学的状態の処置および診断のためのマイクロｒｎａおよび該マイクロｒｎａを含む組成物
JP2017528441A (ja) Ｍｙｈ７ｂの阻害剤およびその使用
AU2024205206A1 (en) Method for managing pain
Enomoto et al. Efficient gene suppression in dorsal root ganglia and spinal cord using adeno-associated virus vectors encoding short-hairpin RNA
WO2017156591A1 (en) Osteoarthritis mirnas
US9309514B2 (en) G protein-coupled purinergic receptor Gpr17 mediates orexigenic effects of FoxO1 in AgRP neurons
McCallister et al. A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD
US20160244755A1 (en) Treating diseases associated with pgc1-alpha by modulating micrornas mir-130a and mir-130b
US20220098592A1 (en) Double stranded rna and uses thereof
CN114206352A (zh) 用于治疗神经变性障碍的组合物和方法
Zhao et al. Reversion of multidrug resistance in human glioma by RNA interference
US20110110896A1 (en) Modulating levels of RNA-binding proteins for the treatment of breast cancer
KR102399851B1 (ko) 인슐린 저항성 검출 및 치료용 조성물 및 방법
JP7804918B2 (ja) ｓｉＲＮＡ、ＤＮＡ、ベクター、女性がん細胞増殖抑制剤、女性がんの腫瘍形成抑制剤、医薬、及び長鎖非コードＲＮＡの利用
US20240026359A1 (en) Methods of Treating of Spinal Stenosis and Ligamentum Flavum Hypertrophy
EP4299742A1 (en) Combination therapy for melanoma
Bailey et al. AAV-mediated gene therapy for SLC13A5 Citrate Transporter Disorder rescues epileptic and metabolic phenotypes
WO2024036343A2 (en) Synergistic nucleic acid based therapeutics and methods of use for treating genetic disorders
JP7137183B2 (ja) がんの治療又は予防用医薬および癌のバイオマーカー
Wang et al. LncRNA DANCR Activates p38/mTOR‐Mediated Autophagy via ANXA2 to Exacerbate Oxygen‐Induced Retinal Neovascularization in Mice

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20100809

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110706

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110902

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20110905

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20120726

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20121026

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130218

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20130710