Invention Grant
- Patent Title: Gene therapy for retinitis pigmentosa
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Application No.: US17383052Application Date: 2021-07-22
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Publication No.: US12201698B2Publication Date: 2025-01-21
- 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
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K9/00 ; A61K31/7105 ; A61K38/17 ; C12N15/113 ; C12N15/86
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
- US20220054657A1 GENE THERAPY FOR RETINITIS PIGMENTOSA Public/Granted day:2022-02-24
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