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Materials and methods for treatment of Usher Syndrome Type 2A
专利权人:
Bayer HealthCare LLC;Crispr Therapeutics AG
发明人:
KANTARDZHIEVA, Albena,NOMA, Akiko,SCARIA, Abraham
申请号:
AU2018393050
公开号:
AU2018393050A1
申请日:
2018.12.21
申请国别(地区):
AU
年份:
2020
代理人:
摘要:
The present application provides materials and methods for treating a patient with Usher Syndrome Type 2A, both ex vivo and in vivo; materials and methods for editing a USH2A gene in a human cell; materials and methods for editing an USH2A gene containing an IVS40 mutation; materials and methods for treating a patient with an USH2A gene containing an IVS40 mutation; and a method for deleting a sequence comprising an IVS40 mutation within a USH2A gene of a cell. The present application also provides one or more gRNAs or sgRNAs for editing an USH2A gene containing an IVS40 mutation. The present application provides a therapeutic for treating a patient with Usher Syndrome Type 2A. The present application also provides a kit for treating a patient with Usher Syndrome Type 2A.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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