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Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
专利权人:
Bayer HealthCare LLC;Crispr Therapeutics AG
发明人:
KANTARDZHIEVA, Albena,NOMA, Akiko,SCARIA, Abraham,TAKEUCHI, Ryo
申请号:
AU2018372763
公开号:
AU2018372763A1
申请日:
2018.11.21
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 autosomal dominant RP, both ex vivo and in vivo; materials and methods for editing a RHO gene in a human cell; and materials and methods for editing a P23H mutation in a RHO gene in a human cell. In addition, the present application provides one or more gRNAs or sgRNAs for editing a RHO gene; one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene. The present application provides a therapeutic for treating a patient with autosomal dominant RP. The present application provides a kit for treating a patient with autosomal dominant RP. In addition, the present application provides a self-inactivating CRISPR-Cas system.
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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