Login processing...

Trial ends in Request Full Access Tell Your Colleague About Jove
JoVE Journal
Medicine

This content is Open Access.

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
 

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

Article doi: 10.3791/53776
May 24th, 2016

Summary May 24th, 2016

Exon skipping is currently a most promising therapeutic option for Duchenne muscular dystrophy (DMD). To expand the applicability for DMD patients and to optimize the stability/function of the resulting truncated dystrophin proteins, a multi-exon skipping approach using cocktail antisense oligonucleotides was developed and we demonstrated systemic dystrophin rescue in a dog model.

Read Article

Get cutting-edge science videos from JoVE sent straight to your inbox every month.

Waiting X
simple hit counter