JP2009509548A5 - - Google Patents
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- JP2009509548A5 JP2009509548A5 JP2008533785A JP2008533785A JP2009509548A5 JP 2009509548 A5 JP2009509548 A5 JP 2009509548A5 JP 2008533785 A JP2008533785 A JP 2008533785A JP 2008533785 A JP2008533785 A JP 2008533785A JP 2009509548 A5 JP2009509548 A5 JP 2009509548A5
- Authority
- JP
- Japan
- Prior art keywords
- shrna
- base pair
- strand portion
- antisense strand
- group
- 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
Links
- 108091027967 Small hairpin RNA Proteins 0.000 claims 26
- 239000004055 small Interfering RNA Substances 0.000 claims 25
- 230000000692 anti-sense effect Effects 0.000 claims 17
- 125000003729 nucleotide group Chemical group 0.000 claims 12
- 239000002773 nucleotide Substances 0.000 claims 8
- 108700028369 Alleles Proteins 0.000 claims 7
- 108091081021 Sense strand Proteins 0.000 claims 5
- 238000000034 method Methods 0.000 claims 5
- 238000012986 modification Methods 0.000 claims 4
- 230000004048 modification Effects 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 4
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims 3
- 230000000875 corresponding effect Effects 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 230000009368 gene silencing by RNA Effects 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 229930010555 Inosine Natural products 0.000 claims 2
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical group O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 2
- 229960003786 inosine Drugs 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 1
- 102100038836 Superoxide dismutase [Cu-Zn] Human genes 0.000 claims 1
- 230000002596 correlated effect Effects 0.000 claims 1
- 229940104302 cytosine Drugs 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000003313 weakening effect Effects 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US11/241,873 US7892793B2 (en) | 2002-11-04 | 2005-09-30 | Allele-specific RNA interference |
| PCT/US2006/038704 WO2007044362A2 (en) | 2005-09-30 | 2006-10-02 | Allele-specific rna interference |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2009509548A JP2009509548A (ja) | 2009-03-12 |
| JP2009509548A5 true JP2009509548A5 (enExample) | 2009-11-26 |
Family
ID=37943327
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008533785A Pending JP2009509548A (ja) | 2005-09-30 | 2006-10-02 | 対立遺伝子特異的ｒｎａ干渉 |
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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7892793B2 (en) * | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| ATE485394T1 (de) * | 2003-06-02 | 2010-11-15 | Univ Massachusetts | Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai |
| AU2004248136B2 (en) | 2003-06-02 | 2011-09-15 | 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 |
| EP2821085B1 (en) * | 2003-09-12 | 2020-04-29 | 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 |
| EP1937066A4 (en) | 2005-08-18 | 2008-12-24 | Alnylam Pharmaceuticals Inc | METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES |
| 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 |
| WO2008018795A1 (en) | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| WO2008143774A2 (en) * | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
| US8841267B2 (en) | 2007-05-23 | 2014-09-23 | Ge Healthcare Dharmacon, Inc. | Micro-RNA scaffolds, non-naturally occurring micro-RNAs, and methods for optimizing non-naturally occurring micro-RNAs |
| AU2008273096B2 (en) | 2007-07-12 | 2013-05-02 | Academisch Ziekenhuis Leiden | Molecules for targeting compounds to various selected organs or tissues |
| CN101790385A (zh) | 2007-07-12 | 2010-07-28 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子 |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| CA2705325C (en) | 2007-11-09 | 2016-11-01 | The Board Of Regents Of The University Of Texas System | Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair |
| EP2249874A1 (en) * | 2008-02-08 | 2010-11-17 | ProSensa Holding BV | Methods and means for treating dna repeat instability associated genetic disorders |
| 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 |
| CN108165548B (zh) | 2008-09-22 | 2022-10-14 | 菲奥医药公司 | 减小大小的自递送RNAi化合物 |
| CA2738625C (en) * | 2008-09-22 | 2017-12-12 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications |
| CN105907756A (zh) * | 2008-12-18 | 2016-08-31 | 戴瑟纳制药公司 | 延长的dicer酶底物和特异性抑制基因表达的方法 |
| WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| US9745574B2 (en) | 2009-02-04 | 2017-08-29 | Rxi Pharmaceuticals Corporation | RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| NZ595955A (en) | 2009-04-24 | 2012-10-26 | Prosensa Technologies Bv | Oligonucleotide comprising an inosine for treating dmd |
| JP6250930B2 (ja) | 2009-05-06 | 2017-12-20 | クルナ・インコーポレーテッド | トリステトラプロリン（ｔｔｐ）に対する天然アンチセンス転写物の抑制によるｔｔｐ関連疾患の治療 |
| CN102459597B (zh) * | 2009-05-08 | 2020-06-30 | 库尔纳公司 | 通过针对dmd家族的天然反义转录物的抑制治疗肌营养蛋白家族相关疾病 |
| US9080171B2 (en) | 2010-03-24 | 2015-07-14 | RXi Parmaceuticals 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 |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| DE18200782T1 (de) * | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
| BR112014026285B1 (pt) | 2012-04-23 | 2021-08-31 | Biomarin Technologies B.V | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| DK3119797T3 (da) | 2014-03-18 | 2021-03-15 | Univ Massachusetts | Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| 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 |
| EP3862005A1 (en) | 2015-07-06 | 2021-08-11 | Phio Pharmaceuticals Corp. | Nucleic acid molecules targeting superoxide dismutase 1 (sod1) |
| CN109414414A (zh) | 2016-03-16 | 2019-03-01 | 戴维·格拉德斯通研究所 | 用于治疗肥胖症和/或糖尿病以及用于鉴定候选治疗剂的方法和组合物 |
| RU2019114169A (ru) * | 2016-10-19 | 2020-11-23 | Девген Нв | Нуклеиновые кислоты, содержащие несовершенные шпильки |
| US10947540B2 (en) * | 2016-11-29 | 2021-03-16 | Association Institut De Myologie | Allele-specific silencing therapy for Dynamin 2-related diseases |
| WO2018152524A1 (en) * | 2017-02-20 | 2018-08-23 | Northwestern University | Toxic rnai active seed sequences for killing cancer cells |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| JP7327803B2 (ja) | 2017-05-09 | 2023-08-16 | 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（ａｌｓ）を処置する方法 |
| CN111448321A (zh) | 2017-09-22 | 2020-07-24 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| AU2019299861A1 (en) | 2018-07-02 | 2021-01-14 | 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 |
| US20230190962A1 (en) * | 2020-02-28 | 2023-06-22 | Blueallele Corporation | Methods for treating gain-of-function disorders combining gene editing and gene therapy |
| EP4499833A4 (en) * | 2022-03-30 | 2025-11-05 | Mirimus Inc | COMPOSITIONS AND METHODS FOR GENERING A NEW AMIARN |
| TW202449167A (zh) | 2023-04-26 | 2024-12-16 | 美商航海家醫療公司 | 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法 |
Family Cites Families (40)
| 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 |
| DE10160151A1 (de) | 2001-01-09 | 2003-06-26 | Ribopharma Ag | Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens |
| 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 |
| EP1309726B2 (en) | 2000-03-30 | 2018-10-03 | Whitehead Institute For Biomedical Research | Rna sequence-specific mediators of rna interference |
| RU2322500C2 (ru) | 2000-12-01 | 2008-04-20 | Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. | Малые молекулы рнк, опосредующие интерференцию рнк |
| WO2003035869A1 (de) | 2001-10-26 | 2003-05-01 | Ribopharma Ag | Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens |
| ES2566561T3 (es) | 2001-07-12 | 2016-04-13 | University Of Massachusetts | Producción in vivo de ARN pequeños de interferencia que median el silenciamiento génico |
| US20030198627A1 (en) | 2001-09-01 | 2003-10-23 | Gert-Jan Arts | siRNA knockout assay method and constructs |
| EP2390328A1 (en) | 2001-09-28 | 2011-11-30 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | MicroRNA molecules |
| US7820632B2 (en) | 2002-02-14 | 2010-10-26 | City Of Hope | Methods for producing interfering RNA molecules in mammalian cells and therapeutic uses for such molecules |
| EP2333062A1 (en) | 2002-07-10 | 2011-06-15 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA-interference by single-stranded RNA molecules |
| US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| ES2280826T5 (es) | 2002-08-05 | 2017-08-03 | Silence Therapeutics Gmbh | Nuevas formas adicionales de moléculas de ARN de interferencia |
| ES2389024T3 (es) | 2002-08-05 | 2012-10-22 | Silence Therapeutics Aktiengesellschaft | Moléculas de RNA interferentes de extremos romos |
| WO2004014933A1 (en) | 2002-08-07 | 2004-02-19 | University Of Massachusetts | Compositions for rna interference and methods of use thereof |
| ATE513843T1 (de) | 2002-09-25 | 2011-07-15 | Univ Massachusetts | Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna |
| ES2334125T3 (es) | 2002-11-04 | 2010-03-05 | University Of Massachusetts | Interferencia de arn especifico de alelos. |
| US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| DK2284266T3 (da) | 2002-11-14 | 2014-01-13 | Thermo Fisher Scient Biosciences Inc | sIRNA-MOLEKYLE MOD TP53 |
| US20040214198A1 (en) | 2002-11-15 | 2004-10-28 | University Of Massachusetts | Allele-targeted RNA interference |
| US20040248299A1 (en) | 2002-12-27 | 2004-12-09 | Sumedha Jayasena | RNA interference |
| DE602004030315D1 (de) | 2003-01-17 | 2011-01-13 | Max Planck Gesellschaft | Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung |
| US20070104688A1 (en) | 2003-02-13 | 2007-05-10 | City Of Hope | Small interfering RNA mediated transcriptional gene silencing in mammalian cells |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| ATE485394T1 (de) * | 2003-06-02 | 2010-11-15 | Univ Massachusetts | Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai |
| AU2004248136B2 (en) | 2003-06-02 | 2011-09-15 | University Of Massachusetts | Methods and compositions for controlling efficacy of RNA silencing |
| JP5654722B2 (ja) | 2003-11-26 | 2015-01-14 | ユニバーシティ オブ マサチューセッツ | 短鎖ｒｎａ機能の配列特異的阻害法 |
| 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 |
| 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 |
| US20050227940A1 (en) | 2004-01-23 | 2005-10-13 | City Of Hope | Amplifying interfering RNA (RNAi) expression and effects |
| US20050256072A1 (en) | 2004-02-09 | 2005-11-17 | 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 |
| US20050273868A1 (en) | 2004-02-17 | 2005-12-08 | University Of Massachusetts | Methods and compositions for enhancing RISC activity in vitro and in vivo |
| WO2005079533A2 (en) | 2004-02-17 | 2005-09-01 | University Of Massachusetts | Methods and compositions for mediating gene silencing |
| 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 |
| JP5243789B2 (ja) | 2004-03-15 | 2013-07-24 | シティ・オブ・ホープ | 二本鎖ｒｎａによる遺伝子発現の特異的阻害のための方法及び組成物 |
| 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 |
-
2005
- 2005-09-30 US US11/241,873 patent/US7892793B2/en active Active
-
2006
- 2006-10-02 JP JP2008533785A patent/JP2009509548A/ja active Pending
- 2006-10-02 AU AU2006302534A patent/AU2006302534A1/en not_active Abandoned
- 2006-10-02 EP EP06836174A patent/EP1940862A4/en not_active Withdrawn
- 2006-10-02 CA CA002624673A patent/CA2624673A1/en not_active Abandoned
- 2006-10-02 WO PCT/US2006/038704 patent/WO2007044362A2/en not_active Ceased
-
2011
- 2011-02-21 US US13/031,534 patent/US8309533B2/en not_active Expired - Lifetime
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