WO2005105995A3 - Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) - Google Patents
Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) Download PDFInfo
- Publication number
- WO2005105995A3 WO2005105995A3 PCT/US2005/006661 US2005006661W WO2005105995A3 WO 2005105995 A3 WO2005105995 A3 WO 2005105995A3 US 2005006661 W US2005006661 W US 2005006661W WO 2005105995 A3 WO2005105995 A3 WO 2005105995A3
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- polyq
- repeat
- diseases
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- C—CHEMISTRY; METALLURGY
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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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
- 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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- General Health & Medical Sciences (AREA)
- Neurosurgery (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP05724244A EP1735443A2 (fr) | 2004-04-14 | 2005-03-01 | Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) |
Applications Claiming Priority (12)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US10/824,036 US20050191638A1 (en) | 2002-02-20 | 2004-04-14 | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
US10/824,036 | 2004-04-14 | ||
US10/826,966 US20050032733A1 (en) | 2001-05-18 | 2004-04-16 | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA) |
US10/826,966 | 2004-04-16 | ||
USPCT/US04/13456 | 2004-04-30 | ||
PCT/US2004/013456 WO2005041859A2 (fr) | 2003-04-30 | 2004-04-30 | Conjugues et compositions |
PCT/US2004/016390 WO2005019453A2 (fr) | 2001-05-18 | 2004-05-24 | Interference arn a mediation assuree par l'inhibition de genes au moyen de petit acide nucleique interferent (ansi) modifie chimiquement |
USPCT/US04/16390 | 2004-05-24 | ||
US10/923,536 US20070042983A1 (en) | 2001-05-18 | 2004-08-20 | RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA) |
US10/923,536 | 2004-08-20 | ||
PCT/US2005/004270 WO2005078097A2 (fr) | 2004-02-10 | 2005-02-09 | Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel) |
USPCT/US05/04270 | 2005-02-09 |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2005105995A2 WO2005105995A2 (fr) | 2005-11-10 |
WO2005105995A3 true WO2005105995A3 (fr) | 2005-12-29 |
Family
ID=56290667
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2005/006661 WO2005105995A2 (fr) | 2004-04-14 | 2005-03-01 | Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) |
Country Status (3)
Country | Link |
---|---|
EP (1) | EP1735443A2 (fr) |
GB (1) | GB2415961A (fr) |
WO (1) | WO2005105995A2 (fr) |
Cited By (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9260716B2 (en) | 2002-08-05 | 2016-02-16 | University Of Iowa Research Foundation | RNA interference suppression of neurodegenerative diseases and methods of use thereof |
US9273356B2 (en) | 2006-05-24 | 2016-03-01 | Medtronic, Inc. | Methods and kits for linking polymorphic sequences to expanded repeat mutations |
US9487779B2 (en) | 2002-08-05 | 2016-11-08 | University Of Iowa Research Foundation | siRNA-mediated gene silencing |
US9528109B2 (en) | 2007-10-26 | 2016-12-27 | Biomarin Technologies B.V. | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA |
Families Citing this family (36)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
US7829694B2 (en) * | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
AU2006210973A1 (en) * | 2005-01-31 | 2006-08-10 | University Of Iowa Research Foundation | Nucleic acid silencing of Huntington's Disease gene |
US20060257912A1 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
JP5425474B2 (ja) | 2006-01-26 | 2014-02-26 | アイシス ファーマシューティカルズ, インコーポレーテッド | ハンチンチン対する、組成物及びその使用 |
GB0605337D0 (en) | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
WO2007123391A1 (fr) | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Intervention thérapeutique dans une maladie génétique chez un individu en modifiant l'expression d'un gène exprimé de manière aberrante. |
EP1857548A1 (fr) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Procédé et moyen permettant d'induire un saut d'exon |
EP2046993A4 (fr) * | 2006-07-07 | 2010-11-17 | Univ Massachusetts | Compositions de silencage de l'arn, et méthodes de traitement de la chorée de huntington |
CA2660523C (fr) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Procedes et moyens de traitement de troubles genetiques associes a l'instabilite des repetitions de l'adn |
US20090036395A1 (en) * | 2007-04-26 | 2009-02-05 | Davidson Beverly L | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
EP2530152B1 (fr) | 2007-05-31 | 2017-12-27 | University of Iowa Research Foundation | Réduction de la toxicité de l'interférence ARN hors cible |
CN101815535B (zh) | 2007-07-12 | 2013-04-24 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官或组织的分子 |
EP2167135A2 (fr) | 2007-07-12 | 2010-03-31 | Prosensa Technologies B.V. | Molécules pour cibler les composants vers plusieurs organes sélectionnés, tissus ou cellules de tumeurs |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
CA2714120A1 (fr) | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methodes et moyens pour traiter les affections genetiques associees a l'instabilite de repetition d'adn |
EP2119783A1 (fr) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes |
EP2317847B1 (fr) | 2008-07-29 | 2019-04-17 | The Board of Regents of The University of Texas System | Inhibition sélective d'expression de protéine de polyglutamine |
US20120046342A1 (en) | 2009-04-24 | 2012-02-23 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
JP5809146B2 (ja) | 2009-09-11 | 2015-11-10 | アイシス ファーマシューティカルズ, インコーポレーテッド | ハンチンチン発現の修飾 |
EP2510098B1 (fr) | 2009-12-09 | 2015-02-11 | Quark Pharmaceuticals, Inc. | Méthodes et compositions utilisées pour le traitement de maladies, d'affections ou de lésions du snc |
WO2012109667A1 (fr) | 2011-02-12 | 2012-08-16 | University Of Iowa Research Foundation | Composés thérapeutiques |
US20150051389A1 (en) | 2011-08-11 | 2015-02-19 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
EP4043039A1 (fr) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Oligonucléotides de modulation arn ayant des caractéristiques améliorées pour le traitement de la dystrophie musculaire de duchenne et becker |
KR102096014B1 (ko) | 2012-12-05 | 2020-04-03 | 알닐람 파마슈티칼스 인코포레이티드 | PCSK9 iRNA 조성물 및 그 사용 방법 |
RU2552913C1 (ru) * | 2014-03-25 | 2015-06-10 | Государственное бюджетное образовательное учреждение высшего профессионального образования "Нижегородская государственная медицинская академия" Министерства Здравоохранения Российской Федерации (ГБОУ ВПО НижГМА Минздрава России) | Способ диагностики болезни гентингтона |
CA2949437C (fr) | 2014-05-20 | 2023-08-15 | University Of Iowa Research Foundation | Composes therapeutiques pour la maladie de huntington |
KR102636633B1 (ko) * | 2014-12-24 | 2024-02-13 | 유니큐어 아이피 비.브이. | 헌팅턴 유전자 억제를 유도하는 RNAi |
WO2016183042A1 (fr) * | 2015-05-10 | 2016-11-17 | Quandx Inc. | Sondes ultrasensibles pour la détection d'acide nucléique |
CN114939124A (zh) | 2015-08-25 | 2022-08-26 | 阿尔尼拉姆医药品有限公司 | 治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物 |
CN113302302A (zh) | 2019-01-09 | 2021-08-24 | 科英布拉大学 | 双链rna及其用途 |
WO2021005223A1 (fr) | 2019-07-10 | 2021-01-14 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Méthodes pour le traitement de l'épilepsie |
EP4149486A1 (fr) * | 2020-05-11 | 2023-03-22 | Genentech, Inc. | Inhibiteurs de la composante c1s du complément pour le traitement d'une maladie neurologique, et compositions associées, systèmes et procédés d'utilisation de ceux-ci |
KR20230162024A (ko) * | 2021-03-29 | 2023-11-28 | 알닐람 파마슈티칼스 인코포레이티드 | 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법 |
Citations (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2003064625A2 (fr) * | 2002-02-01 | 2003-08-07 | Sequitur, Inc. | Compositions oligonucleotidiques presentant une efficacite amelioree |
WO2003070910A2 (fr) * | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Inhibition induite par interference d'arn du facteur de croissance endothelial vasculaire et expression genetique du recepteur de facteur de croissance endothelial vasculaire au moyen d'acides nucleiques interferents courts (sina) |
WO2004013280A2 (fr) * | 2002-08-05 | 2004-02-12 | University Of Iowa Research Foundation | Silençage genique a mediation par arnsi specifique des alleles |
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) |
WO2005078097A2 (fr) * | 2004-02-10 | 2005-08-25 | Sirna Therapeutics, Inc. | Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel) |
Family Cites Families (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20040092465A1 (en) * | 2002-11-11 | 2004-05-13 | Isis Pharmaceuticals Inc. | Modulation of huntingtin interacting protein 1 expression |
JP3917034B2 (ja) * | 2002-07-23 | 2007-05-23 | 湖北工業株式会社 | 光コネクタおよびその製造方法 |
US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
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 |
-
2005
- 2005-03-01 EP EP05724244A patent/EP1735443A2/fr not_active Withdrawn
- 2005-03-01 WO PCT/US2005/006661 patent/WO2005105995A2/fr not_active Application Discontinuation
- 2005-04-06 GB GB0507019A patent/GB2415961A/en not_active Withdrawn
Patent Citations (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2003064625A2 (fr) * | 2002-02-01 | 2003-08-07 | Sequitur, Inc. | Compositions oligonucleotidiques presentant une efficacite amelioree |
WO2003070910A2 (fr) * | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Inhibition induite par interference d'arn du facteur de croissance endothelial vasculaire et expression genetique du recepteur de facteur de croissance endothelial vasculaire au moyen d'acides nucleiques interferents courts (sina) |
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) |
WO2004013280A2 (fr) * | 2002-08-05 | 2004-02-12 | University Of Iowa Research Foundation | Silençage genique a mediation par arnsi specifique des alleles |
WO2005078097A2 (fr) * | 2004-02-10 | 2005-08-25 | Sirna Therapeutics, Inc. | Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel) |
Non-Patent Citations (1)
Title |
---|
LIU W ET AL: "Specific inhibition of Huntington's disease gene expression by siRNAs in cultures cells", PROCEEDINGS OF THE JAPAN ACADEMY. SERIES B, PHYSICAL AND BIOLOGICAL SCIENCES, vol. 79B, no. 10, December 2003 (2003-12-01), pages 293 - 298, XP002983007, ISSN: 0386-2208 * |
Cited By (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9260716B2 (en) | 2002-08-05 | 2016-02-16 | University Of Iowa Research Foundation | RNA interference suppression of neurodegenerative diseases and methods of use thereof |
US9487779B2 (en) | 2002-08-05 | 2016-11-08 | University Of Iowa Research Foundation | siRNA-mediated gene silencing |
US9273356B2 (en) | 2006-05-24 | 2016-03-01 | Medtronic, Inc. | Methods and kits for linking polymorphic sequences to expanded repeat mutations |
US9528109B2 (en) | 2007-10-26 | 2016-12-27 | Biomarin Technologies B.V. | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA |
Also Published As
Publication number | Publication date |
---|---|
WO2005105995A2 (fr) | 2005-11-10 |
EP1735443A2 (fr) | 2006-12-27 |
GB0507019D0 (en) | 2005-05-11 |
GB2415961A (en) | 2006-01-11 |
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