Title

DUX4 Transcript Knockdown with Antisense 2'-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy.

Document Type

Journal Article

Publication Date

2-3-2021

Journal

Molecular Therapy: The Journal of the American Society of Gene Therapy

Volume

29

Issue

2

Inclusive Pages

848-858

DOI

10.1016/j.ymthe.2020.10.010

Keywords

Animals; Disease Models, Animal; Gene Knockdown Techniques; Gene Silencing; Genetic Therapy; Homeodomain Proteins; Humans; Mice; Mice, Knockout; Muscle, Skeletal; Muscular Dystrophy, Facioscapulohumeral; Oligonucleotides, Antisense

Comments

Epub 2020 Oct 15

Peer Reviewed

1

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