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

Authors

Kenji Rowel Q Lim
Adam Bittel
Rika Maruyama
Yusuke Echigoya
Quynh Nguyen
Yiqing Huang
Kasia Dzierlega
Aiping Zhang
Yi-Wen Chen, George Washington University
Toshifumi Yokota

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

APA Citation

Lim, K., Bittel, A., Maruyama, R., Echigoya, Y., Nguyen, Q., Huang, Y., Dzierlega, K., Zhang, A., Chen, Y., & Yokota, T. (2021). DUX4 Transcript Knockdown with Antisense 2'-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy.. Molecular Therapy: The Journal of the American Society of Gene Therapy, 29 (2). http://dx.doi.org/10.1016/j.ymthe.2020.10.010

Peer Reviewed

1