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 |
| ES2712695T3 (es) | 2003-06-02 | 2019-05-14 | Univ Massachusetts | Métodos y composiciones para controlar la eficacia de la silenciación del ARN |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| AU2004252442B2 (en) * | 2003-06-02 | 2010-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of FNAi |
| 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 |
| JP2009504782A (ja) | 2005-08-18 | 2009-02-05 | 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 | 神経疾患を治療するための方法および組成物 |
| US20090018097A1 (en) * | 2005-09-02 | 2009-01-15 | Mdrna, 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 |
| EP2160477B1 (en) * | 2007-05-23 | 2013-08-07 | Dharmacon, Inc. | Micro-RNA scaffolds and non-naturally occurring micro-RNAs |
| CN103212085A (zh) | 2007-07-12 | 2013-07-24 | 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 |
| CA2693742A1 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| EP2203173B1 (en) | 2007-10-26 | 2015-12-23 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| MX2010005166A (es) | 2007-11-09 | 2010-10-07 | Univ Texas | Micro arn de la familia mir-15 que modulan la sobrevivencia de cardiomiocitos y reparacion cardiaca. |
| WO2009099326A1 (en) * | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
| EP2247729B1 (en) * | 2008-02-11 | 2019-05-01 | 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 |
| JP2012504389A (ja) * | 2008-09-22 | 2012-02-23 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法 |
| EP2340309A2 (en) | 2008-09-22 | 2011-07-06 | Rxi Pharmaceuticals Corporation | Neutral nanotransporters |
| EP2379083B1 (en) * | 2008-12-18 | 2017-08-16 | Dicerna Pharmaceuticals, Inc. | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| 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 |
| EP2421971B1 (en) | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| WO2010129746A2 (en) | 2009-05-06 | 2010-11-11 | Curna, Inc. | Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp |
| WO2010129861A2 (en) * | 2009-05-08 | 2010-11-11 | Curna, Inc. | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
| EP2550000A4 (en) | 2010-03-24 | 2014-03-26 | Advirna Inc | RNAI COMPOUNDS OF REDUCED SIZE ADMINISTERING |
| 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 |
| CN118581086A (zh) | 2012-01-27 | 2024-09-03 | 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 |
| WO2013151663A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| EP3604536B1 (en) | 2012-04-23 | 2024-05-29 | Vico Therapeutics B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| CA3276269A1 (en) | 2014-03-18 | 2026-03-02 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | 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 |
| US11001845B2 (en) | 2015-07-06 | 2021-05-11 | Phio Pharmaceuticals Corp. | Nucleic acid molecules targeting superoxide dismutase 1 (SOD1) |
| WO2017161043A1 (en) | 2016-03-16 | 2017-09-21 | The J. David Gladstone Institutes | Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents |
| WO2018072821A1 (en) * | 2016-10-19 | 2018-04-26 | Devgen Nv | Nucleic acids comprising imperfect hairpins |
| 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 |
| SG11201909870SA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US11739330B2 (en) | 2017-09-22 | 2023-08-29 | University Of Massachusetts | SOD1 dual expression vectors and uses thereof |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CA3103963A1 (en) | 2018-07-02 | 2020-01-09 | 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 |
| EP4110406A4 (en) * | 2020-02-28 | 2024-02-28 | Blueallele, LLC | METHOD FOR TREATING GAIN-OF-FUNCTION DISORDERS USING A COMBINATION OF GENERATE 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 |
| 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 |
| US7829693B2 (en) * | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| ATE450621T2 (de) * | 2000-03-30 | 2009-12-15 | Whitehead Biomedical Inst | Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind |
| KR100872437B1 (ko) * | 2000-12-01 | 2008-12-05 | 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. | Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자 |
| WO2003035869A1 (de) | 2001-10-26 | 2003-05-01 | Ribopharma Ag | Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens |
| ES2609014T3 (es) * | 2001-07-12 | 2017-04-18 | University Of Massachusetts | Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico |
| US20030198627A1 (en) | 2001-09-01 | 2003-10-23 | Gert-Jan Arts | siRNA knockout assay method and constructs |
| CA2462144C (en) * | 2001-09-28 | 2016-09-20 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Micro-rna molecules |
| WO2003068797A1 (en) * | 2002-02-14 | 2003-08-21 | 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 |
| 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 |
| DK1389637T3 (da) | 2002-08-05 | 2012-09-03 | Silence Therapeutics Ag | Interfererende RNA-molekyler med stumpe ender |
| WO2004014933A1 (en) * | 2002-08-07 | 2004-02-19 | University Of Massachusetts | Compositions for rna interference and methods of use thereof |
| EP1556402B1 (en) | 2002-09-25 | 2011-06-22 | University of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
| US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| ATE443764T1 (de) | 2002-11-04 | 2009-10-15 | Univ Massachusetts | Allelspezifische rna-interferenz |
| JP2006507841A (ja) * | 2002-11-14 | 2006-03-09 | ダーマコン， インコーポレイテッド | 機能的ｓｉＲＮＡおよび超機能的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 |
| ES2356910T3 (es) * | 2003-01-17 | 2011-04-14 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Construcciones para la expresión inducible de arn de interferencia pequeño (arns) para el silenciamiento génico seleccionado. |
| US20070104688A1 (en) * | 2003-02-13 | 2007-05-10 | City Of Hope | Small interfering RNA mediated transcriptional gene silencing in mammalian cells |
| ES2712695T3 (es) * | 2003-06-02 | 2019-05-14 | Univ Massachusetts | Métodos y composiciones para controlar la eficacia de la silenciación del ARN |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| AU2004252442B2 (en) * | 2003-06-02 | 2010-04-08 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of FNAi |
| EP1793674B1 (en) * | 2003-11-26 | 2018-05-30 | University of Massachusetts | Sequence-specific inhibtion 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 |
| US20060069050A1 (en) * | 2004-02-17 | 2006-03-30 | University Of Massachusetts | Methods and compositions for mediating gene silencing |
| US20050273868A1 (en) * | 2004-02-17 | 2005-12-08 | University Of Massachusetts | Methods and compositions for enhancing RISC activity in vitro and in vivo |
| US8084599B2 (en) * | 2004-03-15 | 2011-12-27 | 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 CA CA002624673A patent/CA2624673A1/en not_active Abandoned
- 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 JP JP2008533785A patent/JP2009509548A/ja active Pending
-
2011
- 2011-02-21 US US13/031,534 patent/US8309533B2/en not_active Expired - Lifetime
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