P28 The antisense oligonucleotide BMN 351 durably ameliorates dystrophic phenotypes in a mouse model of exon 51 –skip-amenable Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is caused by mutation of the DMD gene. Exon skipping with antisense oligonucleotides (ASOs) can be used to restore the DMD open reading frame, enabling production of internally shortened —but still functional—dystrophin. BMN 351 is a next-generation 2’-O-methyl phosphorothioate ASO designed to induce efficient exon 51 skipping by targeting a novel alternative splicing enhancer site. It also contains a 5’ triethylene glycol modification and 5-methyl cytosines to improve ASO s tability and pharmacological characteristics.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: D. Porco, D. Neil, B. Crawford, C. O'Neill, Y. Qi, T. Oppeneer, K. Larimore, S. Gupta, F. Beretta Source Type: research