Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

Facioscapulohumeral muscular dystrophy (FSHD) arises from epigenetic changes that de-repress the DUX4 gene in muscle. The full-length DUX4 protein causes cell death and muscle toxicity, and therefore we hypothesize that FSHD therapies should center on inhibiting full-length DUX4 expression. In this...

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Main Authors: Afrooz Rashnonejad (Author), Gholamhossein Amini-Chermahini (Author), Noah K. Taylor (Author), Nicolas Wein (Author), Scott Q. Harper (Author)
Format: Book
Published: Elsevier, 2021-03-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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