Adeno‐Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines
Our studies suggest that AAV8 and AAV9 transduce fast and slow myofibres at equivalent efficiency. Importantly, uDys therapy effectively prevented dystrophic myofibre atrophy. Our study provides important insight into systemic muscle AAV delivery in large mammals and supports further development of uDys gene therapy for DMD.
Authors
- Dennis O. Pérez‐López (ORCID: https://orcid.org/0000-0001-5504-2409)
- Gang Yao (ORCID: https://orcid.org/0000-0002-2763-8667)
- James Teixeira (ORCID: https://orcid.org/0000-0002-0618-2605)
- Dongsheng Duan (ORCID: https://orcid.org/0000-0003-4109-1132)
- Chady H. Hakim (ORCID: https://orcid.org/0000-0001-6865-050X)
- Roland W. Herzog (ORCID: https://orcid.org/0000-0002-7213-998X)
- Yongping Yue (ORCID: https://orcid.org/0000-0002-1640-2106)
- Xiufang Pan (ORCID: https://orcid.org/0000-0002-2594-5911)
- Matthew J. Burke (ORCID: https://orcid.org/0000-0002-9112-5452)
- Braiden M. Blatt
Institutions
- Missouri College (US)
- Indiana University Indianapolis (US)
- Indiana University – Purdue University Indianapolis (US)
- University of Missouri (US)
Publication Details
- Journal
- Journal of Cachexia Sarcopenia and Muscle
- Published
- 2025-01-10
- DOI
- https://doi.org/10.1002/jcsm.13681
- Citations
- 4
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 3.22
Funders
- National Institutes of Health