Invention Application
- Patent Title: Adeno-Associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease
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Application No.: US17816367Application Date: 2022-07-29
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Publication No.: US20220387560A1Publication Date: 2022-12-08
- Inventor: George Buchlis , Xavier Anguela , Katherine A. High
- Applicant: SPARK THERAPEUTICS, INC.
- Applicant Address: US PA Philadelphia
- Assignee: SPARK THERAPEUTICS, INC.
- Current Assignee: SPARK THERAPEUTICS, INC.
- Current Assignee Address: US PA Philadelphia
- Main IPC: A61K38/46
- IPC: A61K38/46 ; C12N9/22 ; C12N15/10 ; C12N15/864 ; A61K31/7088
Abstract:
Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno-associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.
Information query
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