Invention Grant
- Patent Title: Gene therapy for retinitis pigmentosa
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Application No.: US15127757Application Date: 2015-03-20
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Publication No.: US10383953B2Publication Date: 2019-08-20
- Inventor: Catherine O'Riordan , Matthew Adamowicz
- Applicant: Genzyme Corporation
- Applicant Address: US MA Cambridge
- Assignee: GENZYME CORPORATION
- Current Assignee: GENZYME CORPORATION
- Current Assignee Address: US MA Cambridge
- Agency: Morrison & Foerster LLP
- International Application: PCT/US2015/021896 WO 20150320
- International Announcement: WO2015/143418 WO 20150924
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K31/7105 ; C12N15/86 ; A61K9/00 ; C12N15/113 ; A61K38/17
Abstract:
Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.
Public/Granted literature
- US20170173183A1 GENE THERAPY FOR RETINITIS PIGMENTOSA Public/Granted day:2017-06-22
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