JP2009509548A5 - - Google Patents
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- Publication number
- 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 |
| CA2527958C (en) * | 2003-06-02 | 2014-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of rnai |
| CA2528012C (en) * | 2003-06-02 | 2015-11-24 | University Of Massachusetts | Methods and compositions for controlling efficacy of rna silencing |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| WO2005027980A1 (en) * | 2003-09-12 | 2005-03-31 | University Of Massachusetts | Rna interference for the treatment of gain-of-function disorders |
| US20060134787A1 (en) | 2004-12-22 | 2006-06-22 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
| EP2239328A3 (en) | 2005-08-18 | 2011-01-05 | Alnylam Pharmaceuticals Inc. | Methods and compositions for treating neurological disease |
| WO2007030167A1 (en) * | 2005-09-02 | 2007-03-15 | Nastech Pharmaceutical Company Inc. | Modification of double-stranded ribonucleic acid molecules |
| WO2008016988A1 (en) | 2006-08-01 | 2008-02-07 | Gen-Probe Incorporated | Methods of nonspecific target capture of nucleic acids |
| JP6047270B2 (ja) | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー．フェー． | Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
| WO2008143774A2 (en) * | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
| WO2008147839A1 (en) | 2007-05-23 | 2008-12-04 | Dharmacon, Inc. | Micro-rna scaffolds and non-naturally occurring micro-rnas |
| JP2010533170A (ja) | 2007-07-12 | 2010-10-21 | プロセンサ テクノロジーズ ビー．ブイ． | 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子 |
| WO2009008727A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs or tissues |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| NZ584793A (en) | 2007-10-26 | 2012-05-25 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| WO2009062169A2 (en) | 2007-11-09 | 2009-05-14 | The Board Of Regents Of The University Of Texas System | Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair |
| JP2011510678A (ja) | 2008-02-08 | 2011-04-07 | プロセンサ ホールディング ビーブイ | Ｄｎａ反復不安定性関連遺伝性障害を治療するための方法及び手段 |
| EP3643782A1 (en) * | 2008-02-11 | 2020-04-29 | Phio Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| EP2342340A1 (en) | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| WO2010033225A2 (en) * | 2008-09-22 | 2010-03-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications |
| KR20110110776A (ko) * | 2008-12-18 | 2011-10-07 | 다이서나 파마수이티컬, 인크. | 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법 |
| US9493774B2 (en) | 2009-01-05 | 2016-11-15 | Rxi Pharmaceuticals Corporation | Inhibition of PCSK9 through RNAi |
| WO2010090762A1 (en) | 2009-02-04 | 2010-08-12 | Rxi Pharmaceuticals Corporation | Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| CN102459595A (zh) | 2009-04-24 | 2012-05-16 | 普罗森那技术公司 | 用于治疗dmd的包含肌苷的寡核苷酸 |
| ES2609655T3 (es) | 2009-05-06 | 2017-04-21 | Curna, Inc. | Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP |
| EP2427554B1 (en) * | 2009-05-08 | 2016-11-16 | CuRNA, Inc. | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
| WO2011119852A1 (en) | 2010-03-24 | 2011-09-29 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering rnai compounds |
| US20110306653A1 (en) | 2010-05-14 | 2011-12-15 | Tagcyx Biotechnologies | Stabilization method of functional nucleic acid |
| GB201105137D0 (en) * | 2011-03-28 | 2011-05-11 | Isis Innovation | Therapeutic molecules for use in the suppression of Parkinson's disease |
| WO2013086041A1 (en) * | 2011-12-05 | 2013-06-13 | The Trustees Of Columbia University In The City Of New York | Transcriptome wiring analysis in parkinson's disease and uses thereof |
| CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| WO2013151664A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| PT2841578T (pt) | 2012-04-23 | 2017-08-10 | Biomarin Tech Bv | Resumo |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| US10808247B2 (en) | 2015-07-06 | 2020-10-20 | Phio Pharmaceuticals Corp. | Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach |
| US11001845B2 (en) | 2015-07-06 | 2021-05-11 | Phio Pharmaceuticals Corp. | Nucleic acid molecules targeting superoxide dismutase 1 (SOD1) |
| JP2019515654A (ja) | 2016-03-16 | 2019-06-13 | ザ ジェイ． デヴィッド グラッドストーン インスティテューツ | 肥満及び／又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物 |
| BR112019007719A2 (pt) * | 2016-10-19 | 2019-07-09 | Devgen Nv | ácidos nucleicos compreendendo forquilhas imperfeitas |
| US10947540B2 (en) * | 2016-11-29 | 2021-03-16 | Association Institut De Myologie | Allele-specific silencing therapy for Dynamin 2-related diseases |
| US11613754B2 (en) * | 2017-02-20 | 2023-03-28 | Northwestern University | Toxic RNAi active seed sequences for killing cancer cells |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
| CN118006607A (zh) | 2017-09-22 | 2024-05-10 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| US20210254103A1 (en) | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| US11946047B2 (en) * | 2018-10-23 | 2024-04-02 | Texas Tech University System | Treatment strategies against anthrax by interfering with critical host factors |
| WO2020112736A1 (en) * | 2018-11-30 | 2020-06-04 | Dow Agrosciences Llc | Extension sequences for rna inhibitory molecules |
| WO2021174168A1 (en) * | 2020-02-28 | 2021-09-02 | Blueallele, Llc | Methods for treating gain-of-function disorders combining gene editing and gene therapy |
| WO2023191957A1 (en) * | 2022-03-30 | 2023-10-05 | Mirimus, Inc. | Compositions and methods of generating novel amirna |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
Family Cites Families (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5843641A (en) * | 1993-02-26 | 1998-12-01 | Massachusetts Institute Of Technology | Methods for the daignosis, of familial amyotrophic lateral sclerosis |
| US6506559B1 (en) | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
| US7829693B2 (en) | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| DE10100586C1 (de) | 2001-01-09 | 2002-04-11 | Ribopharma Ag | Verfahren zur Hemmung der Expression eines Ziegens |
| DE10160151A1 (de) | 2001-01-09 | 2003-06-26 | Ribopharma Ag | Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens |
| KR20080023768A (ko) | 2000-03-30 | 2008-03-14 | 화이트헤드 인스티튜트 포 바이오메디칼 리서치 | Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체 |
| CN100523215C (zh) | 2000-12-01 | 2009-08-05 | 马普科技促进协会 | 介导rna干涉的小rna分子 |
| WO2003035869A1 (de) | 2001-10-26 | 2003-05-01 | Ribopharma Ag | Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens |
| BR0211111A (pt) | 2001-07-12 | 2004-06-22 | Univ Massachusetts | Molécula de ácido nucleico isolada, vetor, célula hospedeira, transgene, precursor de rna engenheirado, animal transgênico não humano, e, método de induzir a interferência de ácido ribonucleico de um gene alvo em uma célula |
| US20030198627A1 (en) | 2001-09-01 | 2003-10-23 | Gert-Jan Arts | siRNA knockout assay method and constructs |
| EP2428570B1 (en) | 2001-09-28 | 2018-05-23 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Microrna molecules |
| JP2005527198A (ja) | 2002-02-14 | 2005-09-15 | シティ・オブ・ホープ | 哺乳動物細胞において、干渉ｒｎａ分子を産生する方法、および該干渉ｒｎａ分子の療法的使用 |
| CA2489174C (en) | 2002-07-10 | 2013-02-05 | Thomas Tuschl | Rna-interference by single-stranded rna molecules |
| ES2389024T3 (es) | 2002-08-05 | 2012-10-22 | Silence Therapeutics Aktiengesellschaft | Moléculas de RNA interferentes de extremos romos |
| US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| EP1857547B2 (en) | 2002-08-05 | 2020-12-02 | Silence Therapeutics GmbH | Further novel forms of interfering RNA molecules |
| AU2003261449A1 (en) | 2002-08-07 | 2004-02-25 | Compositions for rna interference and methods of use thereof | |
| WO2004029212A2 (en) | 2002-09-25 | 2004-04-08 | University Of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
| AU2003290586B2 (en) | 2002-11-04 | 2009-07-02 | University Of Massachusetts | Allele-specific RNA interference |
| US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| EP2305813A3 (en) | 2002-11-14 | 2012-03-28 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| WO2004046324A2 (en) | 2002-11-15 | 2004-06-03 | University Of Massachusetts | Allele-targeted rna interference |
| US20040248299A1 (en) | 2002-12-27 | 2004-12-09 | Sumedha Jayasena | RNA interference |
| EP1583832B1 (en) | 2003-01-17 | 2010-12-01 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Inducible small interfering rna (sirna) expression constructs for targeted gene silencing |
| US20070104688A1 (en) | 2003-02-13 | 2007-05-10 | City Of Hope | Small interfering RNA mediated transcriptional gene silencing in mammalian cells |
| CA2528012C (en) | 2003-06-02 | 2015-11-24 | University Of Massachusetts | Methods and compositions for controlling efficacy of rna silencing |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| CA2527958C (en) | 2003-06-02 | 2014-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of rnai |
| US8685946B2 (en) | 2003-11-26 | 2014-04-01 | Universiy of Massachusetts | Sequence-specific inhibition of small RNA function |
| US20060134787A1 (en) | 2004-12-22 | 2006-06-22 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA |
| WO2005062937A2 (en) | 2003-12-22 | 2005-07-14 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna |
| US20050227940A1 (en) | 2004-01-23 | 2005-10-13 | City Of Hope | Amplifying interfering RNA (RNAi) expression and effects |
| WO2005078096A2 (en) | 2004-02-09 | 2005-08-25 | University Of Massachusetts | Dual functional oligonucleotides for use in repressing mutant gene expression |
| US20050182005A1 (en) | 2004-02-13 | 2005-08-18 | Tuschl Thomas H. | Anti-microRNA oligonucleotide molecules |
| US20060069050A1 (en) | 2004-02-17 | 2006-03-30 | University Of Massachusetts | Methods and compositions for mediating gene silencing |
| WO2005079532A2 (en) | 2004-02-17 | 2005-09-01 | University Of Massachusetts | Methods and compositions for enhancing risc activity in vitro and in vivo |
| CA2559955C (en) | 2004-03-15 | 2016-02-16 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded rna |
| US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
| US20060178334A1 (en) | 2005-02-04 | 2006-08-10 | City Of Hope | Double-stranded and single-stranded RNA molecules with 5 ' triphosphates and their use for inducing interferon |
| US20070207874A1 (en) * | 2006-03-03 | 2007-09-06 | Klein Bernard E | Golf ball with optical feedback for improving putting stroke |
-
2005
- 2005-09-30 US US11/241,873 patent/US7892793B2/en active Active
-
2006
- 2006-10-02 WO PCT/US2006/038704 patent/WO2007044362A2/en not_active Ceased
- 2006-10-02 AU AU2006302534A patent/AU2006302534A1/en not_active Abandoned
- 2006-10-02 JP JP2008533785A patent/JP2009509548A/ja active Pending
- 2006-10-02 CA CA002624673A patent/CA2624673A1/en not_active Abandoned
- 2006-10-02 EP EP06836174A patent/EP1940862A4/en not_active Withdrawn
-
2011
- 2011-02-21 US US13/031,534 patent/US8309533B2/en not_active Expired - Lifetime
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