Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells
All these findings suggest challenges other than AON delivery for high level of protein restoration in DMD, highlighting the importance of investigating the biological mechanisms upstream of protein production to further enhance the efficiency of any AON treatment in this condition.
Authors
- Rachele Rossi (ORCID: https://orcid.org/0000-0001-8264-4798)
- Pierpaolo Ala
- Silvia Torelli
- Jennifer E. Morgan (ORCID: https://orcid.org/0000-0002-0375-476X)
- Jyoti Malhotra (ORCID: https://orcid.org/0000-0001-8586-0234)
- Marc T. Moore (ORCID: https://orcid.org/0000-0002-3322-6159)
- Francesco Muntoni
Institutions
- Great Ormond Street Hospital (GB)
- National Institute for Health and Care Research (GB)
- Sarepta Therapeutics (United States) (US)
- University College London (GB)
- Teesside University (GB)
Publication Details
- Journal
- Skeletal Muscle
- Published
- 2024-11-29
- DOI
- https://doi.org/10.1186/s13395-024-00360-4
- Citations
- 5
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.17
Funders
- Sarepta Therapeutics
- National Institute for Health and Care Research
- Medical Research Council