CN108136049A - Galgt2基因治疗的方法和材料 - Google Patents
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| MA45477A (fr) | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| US11338045B2 (en) | 2017-03-17 | 2022-05-24 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| TW202532642A (zh) * | 2017-04-14 | 2025-08-16 | 美商銳進科斯生物股份有限公司 | 使用由人類神經或膠細胞產生的重組人類艾杜糖醛酸鹽 (iduronate)-2-硫酸酯酶 (ids) 之黏多醣病ii之治療 |
| EP3697915A4 (en) | 2017-10-18 | 2021-12-08 | Research Institute at Nationwide Children's Hospital | ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION SPECIFIC TO MUSCLES TO TREAT MUSCLE DYSTROPHY |
| KR20240164969A (ko) | 2017-10-20 | 2024-11-21 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | Nt-3 유전자 치료를 위한 방법 및 물질 |
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| CN120393053A (zh) | 2018-06-18 | 2025-08-01 | 全国儿童医院研究所 | 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送 |
| EP4048286A4 (en) * | 2019-10-25 | 2023-12-06 | Audentes Therapeutics, Inc. | Compositions and methods for treating glycogen storage disorders |
| EP4051324A4 (en) * | 2019-10-28 | 2023-11-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
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| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
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| CA2625279A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
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| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| CN1233291A (zh) | 1996-09-06 | 1999-10-27 | 宾西法尼亚大学托管会 | 重组腺伴随病毒定向基因治疗的方法 |
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| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
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| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
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| US20060194265A1 (en) | 2001-10-23 | 2006-08-31 | Morris David W | Novel therapeutic targets in cancer |
| US20100267575A1 (en) * | 2006-10-17 | 2010-10-21 | Childrens Hospital Medical Center | Gene array technique for predicting response in inflammatory bowel diseases |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| WO2013102904A1 (en) * | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| CA2864879C (en) | 2012-02-17 | 2021-07-20 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
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| HARDUIN-LEPERS, A.: "GenBank: AJ517771.1", 《NCBI》 * |
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