ES2856403T3 - Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica - Google Patents
Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica Download PDFInfo
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- ES2856403T3 ES2856403T3 ES15764861T ES15764861T ES2856403T3 ES 2856403 T3 ES2856403 T3 ES 2856403T3 ES 15764861 T ES15764861 T ES 15764861T ES 15764861 T ES15764861 T ES 15764861T ES 2856403 T3 ES2856403 T3 ES 2856403T3
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| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| WO2011005761A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Novel nucleic acid prodrugs and methods use thereof |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| WO2011133890A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| EP2561075B1 (en) | 2010-04-23 | 2018-06-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| ES2661680T3 (es) | 2011-04-21 | 2018-04-03 | University Of Massachusetts | Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina |
| EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| AU2013288048A1 (en) | 2012-07-13 | 2015-01-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
| KR20230169197A (ko) | 2014-11-14 | 2023-12-15 | 보이저 테라퓨틱스, 인크. | 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법 |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
| US10285388B2 (en) | 2015-05-29 | 2019-05-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a disruption in a C9ORF72 locus |
| ES2978086T3 (es) | 2015-10-22 | 2024-09-05 | Univ Massachusetts | Terapia génica con aspartoacilasa en el tratamiento de enfermedad de Canavan |
| CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
| EP4094780A3 (en) | 2016-02-12 | 2023-02-08 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| EP3432933B1 (en) * | 2016-03-22 | 2022-10-19 | President and Fellows of Harvard College | Biocompatible adhesives and methods of use thereof |
| MX394648B (es) * | 2016-03-31 | 2025-03-24 | Univ Cincinnati | Composiciones que contienen vectores modificados de virus adeno-asociados para usarse en el tratamiento de esclerosis lateral amiotrófica. |
| EP3435755B1 (en) * | 2016-04-02 | 2023-11-01 | Research Institute at Nationwide Children's Hospital | Modified u6 promoter system for tissue specific expression |
| US11207426B2 (en) | 2016-04-05 | 2021-12-28 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| IL297576B2 (en) * | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| CN117904112A (zh) | 2016-05-18 | 2024-04-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2017214471A1 (en) * | 2016-06-10 | 2017-12-14 | Saunders, Ann M. | Methods for detecting structural variants in neurodegenerative disease |
| WO2017218852A1 (en) | 2016-06-15 | 2017-12-21 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| CA3038548A1 (en) * | 2016-09-30 | 2018-04-05 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| KR102695749B1 (ko) | 2016-11-21 | 2024-08-14 | 나노스트링 테크놀로지스, 인크. | 화학적 조성물 및 이것을 사용하는 방법 |
| TWI897844B (zh) * | 2017-05-05 | 2025-09-21 | 美商航海家醫療公司 | 調節多核苷酸 |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | 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 |
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| AU2018352236B2 (en) | 2017-10-16 | 2025-04-10 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
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| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
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