one day more专利名称：AAV-MEDIATED HOMOLOGY-INDEPENDENT TARGETED INTEGRATION GENE EDITING
FOR CORRECTION OF DIVERSE DMD
MUTATIONS IN PATIENTS WITH MUSCULAR
DYSTROPHY
发明人：FLANIGAN, Kevin,STEPHENSON, Anthony, Aaron,HAVENS, Julian
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申请号：US2021/050461
申请日：20210915
公开号：WO2022/060841A2
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公开日：bolted
20220324
专利内容由知识产权出版社提供
摘要：Disclod herein are products, methods, and us for a new gene therapy for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy involving a mutation amenable to DNA repair including, but not limited to, any mutation involving, surrounding, or affecting various regions of the DMD gene. Specifically, the disclosure provides products and methods for fixing diver DMD mutations by replacement of large gments of the DMD gene comprising multiple exons, using CRISPR/Cas9 and Homology-Independent Targeted-Integration (HITI) to accomplish high efficiency knock-in or make large replacements using the non-homologous with or without you
雅思高频词汇end-joining (NHEJ) DNA repair pathway, previously not achievable. In particular, the disclosure provides products, methods and us for the replacement of DMD exons 1-19, 2-19, or 41-55.
申请人：RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
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地址：700 Children's Drive Room W172 Columbus, OH 43205国籍：US
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代理人：JANULIS, Lynn, L.
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