换一批摘要Aspects of the disclosure relate to compositions (e.g., isolated nucleic acids, rAAV vectors, rAAVs, etc.) and methods for gene editing. The disclosure is based, in part, on isolated nucleic acids encoding combinations of gene editing proteins (e.g., Cas proteins) and base editors (e.g., Adenosine Deaminase Acting on RNA deaminase domains) with certain regulatory sequences that are amenable to packaging in recombinant adeno-associated viruses (rAAVs). In some embodiments, compositions described by the disclosure are useful for treating certain diseases in a subject in need thereof.
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申请/专利号
US202117918714A
申请日期
2021-04-13
公开/公告号
公开/公告日
2023-11-02
主分类号
A61K48/0066（含有插入到活体细胞中的遗传物质以治疗遗传病的医药配制品；基因治疗）
分类号
A61K48/0066 C12N15/11 C12N9/22 C12N9/78 C12Y305/04004 C12N15/86 A61P43/00 C12N2310/20 C12N2750/14143
申请/专利权人
University of Massachusetts
发明/设计人
学术成果认领
国别省市代码
美国;US
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