ES2761614T3 - Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto - Google Patents
Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto Download PDFInfo
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- ES2761614T3 ES2761614T3 ES18185820T ES18185820T ES2761614T3 ES 2761614 T3 ES2761614 T3 ES 2761614T3 ES 18185820 T ES18185820 T ES 18185820T ES 18185820 T ES18185820 T ES 18185820T ES 2761614 T3 ES2761614 T3 ES 2761614T3
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| PT3308788T (pt) | 2005-06-23 | 2019-01-30 | Cold Spring Harbor Laboratory | Composições e métodos para a modulação do splicing de smn2 |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| AU2009323766B2 (en) | 2008-12-02 | 2016-10-06 | Wave Life Sciences Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| AU2010262862C1 (en) | 2009-06-17 | 2020-04-30 | Biogen Ma Inc. | Compositions and methods for modulation of SMN2 splicing in a subject |
| SG177564A1 (en) | 2009-07-06 | 2012-02-28 | Ontorii Inc | Novel nucleic acid prodrugs and methods of use thereof |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| KR101829644B1 (ko) | 2010-05-28 | 2018-02-19 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| WO2012031243A2 (en) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS |
| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| DK2638163T3 (en) | 2010-11-12 | 2017-07-24 | Massachusetts Gen Hospital | POLYCOMB-ASSOCIATED NON-CODING RNAs |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| CN103619356B (zh) | 2011-05-05 | 2017-09-12 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| US9833439B2 (en) | 2011-05-25 | 2017-12-05 | Universite Paris Descartes | ERK inhibitors for use in treating spinal muscular atrophy |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| BR112014011018A2 (pt) | 2011-11-11 | 2017-05-02 | Santaris Pharma As | compostos para modulação de splining de smn2 |
| KR20140097398A (ko) | 2011-11-18 | 2014-08-06 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트 |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| CA2858576A1 (en) * | 2011-12-06 | 2013-06-13 | Research Institute At Nationwide Children's Hospital | Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease |
| EP2831232A4 (en) | 2012-03-30 | 2015-11-04 | Univ Washington | METHODS OF MODULATING THE EXPRESSION OF TAU TO REDUCE THE STROKE AND MODIFY A NEURODEGENERATIVE SYMPTOM |
| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ | 不斉補助基 |
| SG10201912895PA (en) | 2012-07-13 | 2020-02-27 | Wave Life Sciences Ltd | Chiral control |
| EP2943225A4 (en) | 2013-01-09 | 2016-07-13 | Ionis Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT |
| WO2014113540A1 (en) * | 2013-01-16 | 2014-07-24 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| KR20150130430A (ko) | 2013-03-14 | 2015-11-23 | 아이시스 파마수티컬즈 인코포레이티드 | 타우 발현을 조절하는 조성물 및 방법 |
| EP3560502B1 (en) | 2013-04-12 | 2025-09-24 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| AU2014292926B2 (en) | 2013-07-25 | 2020-03-05 | Exicure Operating Company | Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use |
| WO2015023797A1 (en) | 2013-08-13 | 2015-02-19 | Northwestern University | Lipophilic nanoparticles for drug delivery |
| WO2015023941A1 (en) | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Oligonucleotides targeting euchromatin regions of genes |
| PL3041958T3 (pl) | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
| WO2015035231A1 (en) | 2013-09-05 | 2015-03-12 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
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