Invention Publication
- Patent Title: NUCLEOTIDE EDITING TO REFRAME DMD TRANSCRIPTS BY BASE EDITING AND PRIME EDITING
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Application No.: US18552262Application Date: 2022-03-25
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Publication No.: US20240165271A1Publication Date: 2024-05-23
- Inventor: Eric N. OLSON , Francesco CHEMELLO , Rhonda BASSEL-DUBY
- Applicant: The Board of Regents of The University of Texas System
- Applicant Address: US TX Austin
- Assignee: The Board of Regents of The University of Texas System
- Current Assignee: The Board of Regents of The University of Texas System
- Current Assignee Address: US TX Austin
- International Application: PCT/US2022/021879 2022.03.25
- Date entered country: 2023-09-25
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K31/7088 ; A61K38/46 ; A61P21/00 ; C07K14/47 ; C12N9/22 ; C12N15/11 ; C12N15/86 ; C12N15/90

Abstract:
Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. Provided herein are methods of using adenine base editor (ABE) to modify splice sites of the dystrophin gene, causing skipping or refraining of common DMD exon deletion mutations, restoring dystrophin expression. Also provided herein are methods of using prime editing to reframe the dystrophin open reading frame and restore dystrophin expression.
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