DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

por Goossens, Remko, Verwey, Nisha, Ariyurek, Yavuz, Schnell, Fred, Aartsma-Rus, Annemieke
Publicado 2023

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

por Lu-Nguyen, Ngoc, Malerba, Alberto, Popplewell, Linda, Schnell, Fred, Hanson, Gunnar, Dickson, George
Publicado 2017