CN119137273A - 包括工程化核内小RNA（snRNA）的组合物和方法 - Google Patents
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| US5814500A (en) | 1996-10-31 | 1998-09-29 | The Johns Hopkins University School Of Medicine | Delivery construct for antisense nucleic acids and methods of use |
| US6692910B2 (en) | 1997-07-28 | 2004-02-17 | The University Of Connecticut | Inhibition of a target messenger RNA with a modified U1 small nuclear RNA |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002083908A1 (es) | 2001-03-30 | 2002-10-24 | Fundacion Para La Investigacion Medica Aplicada | Procedimiento de inhibicion reversible de la expresion genica mediante ribonucleoproteinas modificadas |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| US8822168B2 (en) | 2005-03-11 | 2014-09-02 | The Trustees Of The University Of Pennsylvania | Assays for detecting small nuclear ribonucleoprotein particle assembly and survival of motor neurons activity |
| US9040491B2 (en) | 2006-08-30 | 2015-05-26 | The Trustees Of The University Of Pennsylvania | Compositions, methods and kits based on small nuclear RNAs |
| US8343941B2 (en) | 2007-03-30 | 2013-01-01 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
| US9441221B2 (en) | 2007-03-30 | 2016-09-13 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
| EP2142672B1 (en) | 2007-03-30 | 2012-09-05 | Rutgers, The State University of New Jersey | Compositions and methods for gene silencing |
| US8907075B2 (en) | 2007-03-30 | 2014-12-09 | Samuel Ian Gunderson | Compositions and methods for gene silencing |
| ES2714007T3 (es) | 2007-04-09 | 2019-05-24 | Univ Florida | Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso |
| US9080170B2 (en) | 2010-03-17 | 2015-07-14 | Association Institut De Myologie | Modified U7 snRNAs for treatment of neuromuscular diseases |
| IT1400425B1 (it) | 2010-06-08 | 2013-05-31 | Amsterdam Molecular Therapeutics Bv | Modified snrnas for use in therapy. |
| IT1405793B1 (it) | 2010-10-15 | 2014-01-24 | Univ Ferrara | Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica |
| US9303258B2 (en) | 2011-04-05 | 2016-04-05 | The Regents Of The University Of California | Method and compositions comprising small RNA agonist and antagonists to modulate inflammation |
| MX388221B (es) | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| CA2957661A1 (en) | 2014-08-09 | 2016-02-18 | Kevin FLANIGAN | Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene |
| US9669109B1 (en) | 2015-11-24 | 2017-06-06 | Universita Di Ferrara | Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| JP6966463B2 (ja) | 2016-02-26 | 2021-11-17 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 組換えウイルス産物及びｄｕｘ４エクソンスキッピングを誘導するための方法 |
| JP2021534755A (ja) | 2018-08-22 | 2021-12-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| CN113924364A (zh) | 2019-03-08 | 2022-01-11 | 罗格斯新泽西州立大学 | 治疗亨廷顿病的组合物和方法 |
| CA3134683A1 (en) | 2019-03-26 | 2020-10-01 | Rutgers, The State University Of New Jersey | Compositions and methods for treating neurodegenerative disorders |
| WO2020214763A1 (en) * | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| US20220348958A1 (en) | 2019-07-23 | 2022-11-03 | Oxford Biomedica (Uk) Limited | Enhancing Production of Lentiviral Vectors |
| WO2021026075A1 (en) | 2019-08-02 | 2021-02-11 | Research Institute At Nationwide Children's Hospital | Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies |
| US20230059753A1 (en) | 2019-12-09 | 2023-02-23 | Astellas Pharma Inc. | Antisense guide rna with added functional region for editing target rna |
| CN115777020A (zh) * | 2020-04-22 | 2023-03-10 | 塑造治疗公司 | 使用snrna组分的组合物和方法 |
| AU2021349277A1 (en) | 2020-09-28 | 2023-05-11 | Research Institute At Nationwide Children's Hospital | Products and methods for treating muscular dystrophy |
| IL303230A (en) | 2020-11-30 | 2023-07-01 | Res Inst Nationwide Childrens Hospital | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
| AU2022262420A1 (en) | 2021-04-23 | 2023-11-16 | Research Institute At Nationwide Children's Hospital | Products and methods for treating muscular dystrophy |
| AU2023228712A1 (en) | 2022-03-04 | 2024-10-17 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising engineered small nuclear rna (snrna) |
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