Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO

Splice modulation using antisense oligonucleotides (AOs) has been shown to yield targeted exon exclusion to restore the open reading frame and generate truncated but partially functional dystrophin protein. This has been successfully demonstrated in dystrophin-deficient mdx mice and in Duchenne musc...

Полное описание

Библиографические подробности
Главные авторы: Yin, H, Moulton, H, Betts, C, Merritt, T, Seow, Y, Ashraf, S, Wang, Q, Boutilier, J, Wood, MJA
Формат: Journal article
Язык:English
Опубликовано: Cell Press 2010