Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics

por Chen, Jennifer CJ, King, Oliver D, Zhang, Yuanfan, Clayton, Nicholas P, Spencer, Carrie, Wentworth, Bruce M, Emerson, Charles P, Wagner, Kathryn R
Publicado 2016

A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

por Gan, Li, Wu, Leslie C.L., Wood, Jenna A., Yao, Monica, Treleaven, Chris M., Estrella, Nelsa L., Wentworth, Bruce M., Hanson, Gunnar J., Passini, Marco A.
Publicado 2022

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

por Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.
Publicado 2012

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

por Clayton, Nicholas P, Nelson, Carol A, Weeden, Timothy, Taylor, Kristin M, Moreland, Rodney J, Scheule, Ronald K, Phillips, Lucy, Leger, Andrew J, Cheng, Seng H, Wentworth, Bruce M
Publicado 2014