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MATERIALS AND METHODS FOR TREATMENT OF USHER SYNDROME TYPE 2A AND/OR NON-SYNDROMIC AUTOSOMAL RECESSIVE RETINITIS PIGMENTOSA (ARRP)
专利权人:
CRISPR THERAPEUTICS AG;BAYER HEALTHCARE LLC
发明人:
Mariacarmela ALLOCCA,Akiko NOMA,Abraham SCARIA
申请号:
US16905258
公开号:
US20200318108A1
申请日:
2020.06.18
申请国别(地区):
US
年份:
2020
代理人:
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.
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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