- 专利标题： Gene Therapy For Eye Pathologies
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申请号： US17600377申请日： 2020-04-02
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公开(公告)号： US20220143221A1公开(公告)日： 2022-05-12
- 发明人: Olivier Danos , Sherri Van Everen , Jesse I. Yoo , Samir Maganbhai Patel , Avanti Arvind Ghanekar , Anthony Ray O'Berry , Kim Rees Irwin-Pack , Darin Thomas Curtiss
- 申请人： REGENXBIO, Inc.
- 申请人地址： US MD Rockville
- 专利权人： REGENXBIO, Inc.
- 当前专利权人： REGENXBIO, Inc.
- 当前专利权人地址： US MD Rockville
- 国际申请： PCT/US2020/026356 WO 20200402
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; A61P27/02
摘要：
Compositions and methods are described for the delivery of therapeutic products (such as therapeutic proteins (for example, antibodies), therapeutic RNAs (for example, shRNAs, siRNAs, and miRNAs), and therapeutic aptamers) to the retina/vitreal humour in the eyes of human subjects to treat pathologies of the eye, involving, for example, recombinant viral vectors such as recombinant adeno-associated virus (rAAV) vectors.
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