Cargando…
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the structural integrity of muscle. As variants in the dystrophin gene lead to a disruption of the reading frame, pharmacologica...
| Autores principales: | , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer International Publishing
2021
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8440545/ https://www.ncbi.nlm.nih.gov/pubmed/33564172 http://dx.doi.org/10.1038/s41431-021-00811-2 |