Co‐Opting <scp>MBNL</scp> ‐Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy
DMX<sup>on</sup> control elements can increase the therapeutic window of viral-based therapeutics in DM1, and activity is dependent upon delivered cargo and model severity. ANN NEUROL 2026;99:211-222.
Authors
- Ellie M. Carrell (ORCID: https://orcid.org/0000-0001-5222-5437)
- Beverly L. Davidson (ORCID: https://orcid.org/0000-0002-3715-6290)
- Samuel Carrell (ORCID: https://orcid.org/0000-0002-0114-495X)
- Ryan C Giovenco
Institutions
- Children's Hospital of Philadelphia (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- Annals of Neurology
- Published
- 2025-08-29
- DOI
- https://doi.org/10.1002/ana.78024
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Children's Hospital of Philadelphia