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

• Κύριοι συγγραφείς: Afrooz Rashnonejad (Συγγραφέας), Gholamhossein Amini-Chermahini (Συγγραφέας), Noah K. Taylor (Συγγραφέας), Nicolas Wein (Συγγραφέας), Scott Q. Harper (Συγγραφέας)
• Μορφή: Βιβλίο
• Έκδοση: Elsevier, 2021-03-01T00:00:00Z.
• Θέματα: article
• Διαθέσιμο Online: Connect to this object online.

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...