Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping

por Rok, Matthew, Wong, Tatianna Wai Ying, Maino, Eleonora, Ahmed, Abdalla, Yang, Grace, Hyatt, Elzbieta, Lindsay, Kyle, Fatehi, Sina, Marks, Ryan, Delgado-Olguín, Paul, Ivakine, Evgueni A., Cohn, Ronald D.
Publicado 2023

Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression

por Maino, Eleonora, Wojtal, Daria, Evagelou, Sonia L, Farheen, Aiman, Wong, Tatianna W Y, Lindsay, Kyle, Scott, Ori, Rizvi, Samar Z, Hyatt, Elzbieta, Rok, Matthew, Visuvanathan, Shagana, Chiodo, Amanda, Schneeweiss, Michelle, Ivakine, Evgueni A, Cohn, Ronald D
Publicado 2021