- 专利标题: AAV vectors for treatment of dominant retinitis pigmentosa
-
申请号: US16081307申请日: 2017-03-01
-
公开(公告)号: US11118185B2公开(公告)日: 2021-09-14
- 发明人: Alfred S. Lewin , William W. Hauswirth , Michael T. Massengill , William Beltran , Gustavo D. Aguirre , Artur Cideciyan , Samuel Jacobson
- 申请人: University of Florida Research Foundation, Incorporated , The Trustees of the University of Pennsylvania
- 申请人地址: US FL Gainesville; US PA Philadelphia
- 专利权人: University of Florida Research Foundation, Incorporated,The Trustees of the University of Pennsylvania
- 当前专利权人: University of Florida Research Foundation, Incorporated,The Trustees of the University of Pennsylvania
- 当前专利权人地址: US FL Gainesville; US PA Philadelphia
- 代理机构: Wolf, Greenfield & Sacks, P.C.
- 国际申请: PCT/US2017/020289 WO 20170301
- 国际公布: WO2017/151823 WO 20170908
- 主分类号: C12N15/86
- IPC分类号: C12N15/86 ; C12N15/113 ; A61P27/02 ; C12N7/00
摘要:
Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.
公开/授权文献
信息查询
IPC分类: