FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?

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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Bibliographic Details
Main Authors:	Charis L. Himeda, Peter L. Jones
Format:	Article
Language:	English
Published:	MDPI AG 2022-05-01
Series:	Journal of Personalized Medicine
Subjects:	facioscapulohumeral muscular dystrophy FSHD DUX4 skeletal muscle muscular dystrophy gene therapy
Online Access:	https://www.mdpi.com/2075-4426/12/6/865

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