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 (https=) | 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 (https=) |
| EP (1) | EP1940862A4 (https=) |
| JP (1) | JP2009509548A (https=) |
| AU (1) | AU2006302534A1 (https=) |
| CA (1) | CA2624673A1 (https=) |
| WO (1) | WO2007044362A2 (https=) |
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 |
| US7750144B2 (en) * | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| EP1633890B2 (en) * | 2003-06-02 | 2020-11-18 | University of Massachusetts | METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi |
| PT1633767T (pt) * | 2003-06-02 | 2019-02-27 | Univ Massachusetts | Métodos e composições para controlar a eficácia do silenciamento de arn |
| DK2821085T3 (da) * | 2003-09-12 | 2020-08-03 | Univ Massachusetts | Rna-interferens til behandling af "gain-of-function"-forstyrrelser |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | 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 |
| US9051601B2 (en) | 2006-08-01 | 2015-06-09 | Gen-Probe Incorporated | Methods of nonspecific target capture of nucleic acids |
| SI2049664T1 (sl) | 2006-08-11 | 2012-04-30 | Prosensa Technologies Bv | Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev |
| WO2008143774A2 (en) * | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
| WO2008147837A1 (en) * | 2007-05-23 | 2008-12-04 | Dharmacon, Inc. | Micro-rna scaffolds, non-naturally occurring micro-rnas, and methods for optimizing non-naturally occurring micro-rnas |
| CA2693742A1 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| WO2009008727A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs or tissues |
| ES2639852T3 (es) | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| KR20100099158A (ko) | 2007-11-09 | 2010-09-10 | 보드 오브 리전츠 더 유니버시티 오브 텍사스 시스템 | 심근세포 생존과 심장 회복을 제어하는 ＭＩＲ-15 집단의 마이크로ＲＮＡｓ |
| WO2009099326A1 (en) * | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
| CA2715289C (en) | 2008-02-11 | 2019-12-24 | Rxi Pharmaceuticals Corporation | 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 |
| DK2341943T3 (en) * | 2008-09-22 | 2019-02-25 | Dicerna Pharmaceuticals Inc | COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS |
| EP2342340A1 (en) | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| KR101728655B1 (ko) * | 2008-12-18 | 2017-04-19 | 다이서나 파마수이티컬, 인크. | 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법 |
| 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 |
| EP2421971B1 (en) | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| 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 |
| JP5931720B2 (ja) * | 2009-05-08 | 2016-06-08 | クルナ・インコーポレーテッド | Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療 |
| RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| 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 |
| US20140323581A1 (en) * | 2011-12-05 | 2014-10-30 | 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 |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| LT2841578T (lt) | 2012-04-23 | 2017-09-25 | Biomarin Technologies B.V. | Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| CA2942515C (en) | 2014-03-18 | 2025-12-09 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| 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 |
| US20200181611A1 (en) * | 2016-10-19 | 2020-06-11 | Devgen Nv | Nucleic acids comprising imprefect hairpins |
| US10947540B2 (en) * | 2016-11-29 | 2021-03-16 | Association Institut De Myologie | Allele-specific silencing therapy for Dynamin 2-related diseases |
| WO2018152523A1 (en) * | 2017-02-20 | 2018-08-23 | Northwestern University | Use of trinucleotide repeat rnas to treat cancer |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| US11859179B2 (en) | 2017-05-09 | 2024-01-02 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (ALS) |
| CN111448321A (zh) | 2017-09-22 | 2020-07-24 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | 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 |
| ES2336887T5 (es) * | 2000-03-30 | 2019-03-06 | Whitehead Inst Biomedical Res | Mediadores de interferencia por ARN específicos de secuencias de ARN |
| CA2429814C (en) * | 2000-12-01 | 2014-02-18 | Thomas Tuschl | Rna interference mediating small rna molecules |
| WO2003035869A1 (de) | 2001-10-26 | 2003-05-01 | Ribopharma Ag | Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens |
| IL159756A0 (en) * | 2001-07-12 | 2004-06-20 | Univ Massachusetts | IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING |
| US20030198627A1 (en) | 2001-09-01 | 2003-10-23 | Gert-Jan Arts | siRNA knockout assay method and constructs |
| EP2447370B1 (en) * | 2001-09-28 | 2018-07-18 | 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 |
| US8101348B2 (en) * | 2002-07-10 | 2012-01-24 | Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. | RNA-interference by single-stranded RNA molecules |
| DE60310944T3 (de) * | 2002-08-05 | 2017-08-03 | Silence Therapeutics Gmbh | Weitere neue formen von interferierende rns moleküle |
| DK1389637T3 (da) | 2002-08-05 | 2012-09-03 | Silence Therapeutics Ag | Interfererende RNA-molekyler med stumpe ender |
| US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| AU2003261449A1 (en) * | 2002-08-07 | 2004-02-25 | Compositions for rna interference and methods of use thereof | |
| CA2881743A1 (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 |
| AU2003295600A1 (en) * | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| AU2003295539A1 (en) | 2002-11-15 | 2004-06-15 | University Of Massachusetts | Allele-targeted rna interference |
| US20040248299A1 (en) * | 2002-12-27 | 2004-12-09 | Sumedha Jayasena | RNA interference |
| EP2314687B1 (en) * | 2003-01-17 | 2017-12-27 | 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 |
| EP1633890B2 (en) | 2003-06-02 | 2020-11-18 | University of Massachusetts | METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi |
| PT1633767T (pt) * | 2003-06-02 | 2019-02-27 | Univ Massachusetts | Métodos e composições para controlar a eficácia do silenciamento de arn |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| GB2424887B (en) * | 2003-11-26 | 2008-05-21 | Univ Massachusetts | Sequence-specific inhibition of small RNA function |
| 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 |
| WO2005079532A2 (en) | 2004-02-17 | 2005-09-01 | University Of Massachusetts | Methods and compositions for enhancing risc activity in vitro and in vivo |
| US20060069050A1 (en) * | 2004-02-17 | 2006-03-30 | 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 |
| 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 |
| 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 AU AU2006302534A patent/AU2006302534A1/en not_active Abandoned
- 2006-10-02 CA CA002624673A patent/CA2624673A1/en not_active Abandoned
- 2006-10-02 WO PCT/US2006/038704 patent/WO2007044362A2/en not_active Ceased
- 2006-10-02 JP JP2008533785A patent/JP2009509548A/ja active Pending
- 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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