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IMPROVED RAAV VECTORS AND METHODS FOR TRANSDUCTION OF PHOTORECEPTORS AND RPE CELLS
专利权人:
University of Florida Research Foundation; Inc.
发明人:
BOYE, Shannon, E.,BOYE, Sanford, L.,AGBANDJE-MCKENNA, Mavis,SRIVASTAVA, Arun
申请号:
EP15758026
公开号:
EP3113787A4
申请日:
2015.03.04
申请国别(地区):
EP
年份:
2017
代理人:
摘要:
Disclosed are capsid-modified rAAV particles and expression vectors, as well as compositions and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and using novel capsid-protein-mutated particle or rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for subretinal delivery of therapeutic gene constructs to mammalian photoreceptors and retinal pigment epithelial cells, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of a variety of inherited retinal diseases.
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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