- 专利标题： Materials and methods for treatment of Usher syndrome type 2A and/or non-syndromic autosomal recessive retinitis pigmentosa (ARRP)
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申请号： US16905258申请日： 2020-06-18
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公开(公告)号： US11118177B2公开(公告)日： 2021-09-14
- 发明人: Mariacarmela Allocca , Akiko Noma , Abraham Scaria
- 申请人： CRISPR THERAPEUTICS AG , BAYER HEALTHCARE LLC
- 申请人地址： CH Zug; US NJ Whippany
- 专利权人： CRISPR THERAPEUTICS AG,BAYER HEALTHCARE LLC
- 当前专利权人： CRISPR THERAPEUTICS AG,BAYER HEALTHCARE LLC
- 当前专利权人地址： CH Zug; US NJ Whippany
- 代理机构： Marshall, Gerstein & Borun LLP
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; A61K35/12 ; A61K48/00 ; A61P27/02 ; C12N9/22 ; C12N15/86 ; A61K38/00
摘要：
The present application provides materials and methods for treating a patient with one or more of Usher Syndrome Type 2A and ARRP, both ex vivo and in vivo; materials and methods for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299. In addition, the present application provides one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; a therapeutic comprising at least one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; and a therapeutic for treating a patient with one or more of Usher Syndrome Type 2A and ARRP. The present application also provides a kit for treating a patient with one or more of Usher Syndrome Type 2A and ARRP.
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