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
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- 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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- 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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- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (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 | 2004-04-14 | ||
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) |
US10/826,966 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 | ||
PCT/US2004/013456 WO2005041859A2 (fr) | 2003-04-30 | 2004-04-30 | Conjugues et compositions |
USPCT/US04/13456 | 2004-04-30 | ||
USPCT/US04/16390 | 2004-05-24 | ||
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 |
US10/923,536 | 2004-08-20 | ||
US10/923,536 US20070042983A1 (en) | 2001-05-18 | 2004-08-20 | RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA) |
USPCT/US05/04270 | 2005-02-09 | ||
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) |
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 |
WO2008005562A2 (fr) * | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Compositions de silençage de l'arn, et méthodes de traitement de la chorée de huntington |
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 |
EP1885854B1 (fr) | 2005-05-06 | 2012-10-17 | Medtronic, Inc. | Procedes et sequences permettant de supprimer l'expression du gene de huntington chez les primates |
US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
US20070299027A1 (en) | 2006-01-26 | 2007-12-27 | Gene Hung | Compositions and their uses directed to huntingtin |
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 |
EP2049664B1 (fr) | 2006-08-11 | 2011-09-14 | Prosensa Technologies B.V. | Oligonucléotides monocaténaires complémentaires des elements repetitifs pour le traitement de troubles génétiques associés a l'instabilité des répétitions 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 |
ES2549122T3 (es) | 2007-05-31 | 2015-10-23 | University Of Iowa Research Foundation | Reducción de toxicidad por interferencia de ARN desviada de su diana |
WO2009008725A2 (fr) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molécules pour cibler des composés vers divers organes, tissus ou cellules tumorales sélectionnés |
NZ582521A (en) | 2007-07-12 | 2011-09-30 | Prosensa Technologies Bv | A conjugate comprising the amino acid sequence LGAQSNF for targeting compounds to muscle tissue |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
NZ587178A (en) | 2008-02-08 | 2011-11-25 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
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 |
WO2010123369A1 (fr) | 2009-04-24 | 2010-10-28 | Prosensa Technologies B.V. | Oligonucléotide comprenant une inosine pour traiter une dystrophie musculaire de duchenne (dmd) |
KR102173836B1 (ko) | 2009-09-11 | 2020-11-05 | 아이오니스 파마수티컬즈, 인코포레이티드 | 헌팅틴 발현의 조절 |
US8778904B2 (en) | 2009-12-09 | 2014-07-15 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the CNS |
WO2012109667A1 (fr) | 2011-02-12 | 2012-08-16 | University Of Iowa Research Foundation | Composés thérapeutiques |
US9752142B2 (en) | 2011-08-11 | 2017-09-05 | Ionis Pharmaceuticals, Inc. | Gapped oligomeric compounds comprising 5′-modified deoxyribonucleosides in the gap and uses thereof |
CN104203289B (zh) | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
JP6574383B2 (ja) | 2012-12-05 | 2019-09-11 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | PCSK9iRNA組成物及びその使用方法 |
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 |
EP3146051B8 (fr) | 2014-05-20 | 2019-11-27 | University of Iowa Research Foundation | Composés thérapeutiques pour la maladie de huntington |
LT3237618T (lt) | 2014-12-24 | 2019-07-10 | Uniqure Ip B.V. | Rnri sukeltas hantingtino geno slopinimas |
CN108138221B (zh) * | 2015-05-10 | 2021-11-05 | 康代思锐公司 | 用于检测核酸的超高灵敏度探针 |
CN114469984A (zh) | 2015-08-25 | 2022-05-13 | 阿尔尼拉姆医药品有限公司 | 用于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 |
WO2020144611A1 (fr) | 2019-01-09 | 2020-07-16 | Universidade De Coimbra | Arn double brin et utilisations correspondantes |
US20220251567A1 (en) | 2019-07-10 | 2022-08-11 | Inserm (Institut National De La Santè Et De La Recherche Médicale) | Methods for the treatment of epilepsy |
JP2023527684A (ja) * | 2020-05-11 | 2023-06-30 | ジェネンテック, インコーポレイテッド | 神経疾患を治療するための補体成分c1s阻害剤、並びにそれを使用する関連組成物、システム及び方法 |
AR125230A1 (es) * | 2021-03-29 | 2023-06-28 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO |
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 |
EP2821085B1 (fr) * | 2003-09-12 | 2020-04-29 | University of Massachusetts | Interférence arn pour le traitement de troubles à gain |
-
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 |
---|---|
GB2415961A (en) | 2006-01-11 |
EP1735443A2 (fr) | 2006-12-27 |
WO2005105995A2 (fr) | 2005-11-10 |
GB0507019D0 (en) | 2005-05-11 |
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