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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...

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Detalles Bibliográficos
Autores principales:	Himeda, Charis L., Jones, Peter L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Review
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

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