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FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?
Facioscapulohumeral muscular dystrophy (FSHD) is arguably one of the most challenging genetic diseases to understand and treat. The disease is caused by epigenetic dysregulation of a macrosatellite repeat, either by contraction of the repeat or by mutations in silencing proteins. Both cases lead to...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9225474/ https://www.ncbi.nlm.nih.gov/pubmed/35743650 http://dx.doi.org/10.3390/jpm12060865 |