Modified MTM1 genes and uses thereof
TW202627081APending Publication Date: 2026-07-01REGENERON PHARMACEUTICALS INC
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Patent Information
- Application Number
- TW114130588
- Authority / Receiving Office
- TW · TW
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-11-21
- Filing Date
- 2025-08-11
- Publication Date
- 2026-07-01
Abstract
Provided herein are modified nucleic acids comprising modified MTM1 genes, modified muscle-specific promoters, e.g., MHCK7, or a combination thereof. Also provided herein are expression cassettes, AAV vector genomes, and AAV particles comprising the same. Further provided herein are methods of delivering MTM1 to a muscle cell and methods of treating XLMTM.
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