CA3079437A1 - Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof - Google Patents
Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof Download PDFInfo
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- CA3079437A1 CA3079437A1 CA3079437A CA3079437A CA3079437A1 CA 3079437 A1 CA3079437 A1 CA 3079437A1 CA 3079437 A CA3079437 A CA 3079437A CA 3079437 A CA3079437 A CA 3079437A CA 3079437 A1 CA3079437 A1 CA 3079437A1
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- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12N15/09—Recombinant DNA-technology
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- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- A01K2267/03—Animal model, e.g. for test or diseases
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| PCT/US2018/056271 WO2019079437A1 (en) | 2017-10-18 | 2018-10-17 | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING THE FUNCTION OF THE HBB GENE AND METHODS OF USE |
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| CN109536494A (zh) * | 2017-10-31 | 2019-03-29 | 广东赤萌医疗科技有限公司 | 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒 |
| WO2021138559A1 (en) * | 2019-12-31 | 2021-07-08 | Swanbio Therapeutics Limited | Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) |
| CN111363756B (zh) * | 2020-04-02 | 2023-01-17 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 一种珠蛋白基因双表达慢病毒载体及其应用 |
| JP2023522852A (ja) * | 2020-04-06 | 2023-06-01 | ホモロジー・メディシンズ・インコーポレイテッド | Ｉｄｓ遺伝子導入のためのアデノ随伴ウイルス組成物及びその使用方法 |
| WO2023019185A1 (en) * | 2021-08-10 | 2023-02-16 | Gentibio, Inc. | Compositions and methods for engineering stable tregs |
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| US5449759A (en) * | 1987-05-16 | 1995-09-12 | Somatogen, Inc. | Hemoglobins with intersubunit desulfide bonds |
| US5252479A (en) * | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| WO1998024924A1 (en) * | 1996-12-05 | 1998-06-11 | Introgene B.V. | Genetic modification of primate hemopoietic repopulating stem cells |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| CN101348786B (zh) * | 2007-07-20 | 2011-05-11 | 谭孟群 | 一种人β珠蛋白基因及其重组腺相关病毒载体 |
| WO2009013559A1 (en) * | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| US11242525B2 (en) * | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| CN114480393A (zh) | 2014-09-04 | 2022-05-13 | 纪念斯隆-凯特琳癌症中心 | 用于治疗血红蛋白病的球蛋白基因治疗 |
| SG11201702073XA (en) | 2014-09-24 | 2017-04-27 | Hope City | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| WO2016134338A1 (en) * | 2015-02-19 | 2016-08-25 | University Of Florida Research Foundation, Inc. | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
| CN109476706A (zh) * | 2016-02-16 | 2019-03-15 | 耶鲁大学 | 用于促进靶向基因编辑的组合物及其使用方法 |
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| AU2018352855B2 (en) | 2022-07-14 |
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