Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy [Medical Sciences]

Facioscapulohumeral muscular dystrophy (FSHD), characterized by progressive muscle weakness and deterioration, is genetically linked to aberrant expression of DUX4 in muscle. DUX4, in its full-length form, is cytotoxic in nongermline tissues. Here, we designed locked nucleic acid (LNA) gapmer antisense oligonucleotides (AOs) to knock down DUX4 in immortalized FSHD myoblasts...

http://www.pnas.org/content/117/28/16509.short?rss=1

Source: Proceedings of the National Academy of Sciences - July 13, 2020 Category: Science Authors: Kenji Rowel Q. Lim, Rika Maruyama, Yusuke Echigoya, Quynh Nguyen, Aiping Zhang, Hunain Khawaja, Sreetama Sen Chandra, Takako Jones, Peter Jones, Yi-Wen Chen, Toshifumi Yokota Tags: Biological Sciences Source Type: research