DMD – ANIMAL MODELS & PRECLINICAL TREATMENT

0

The gene corrective therapy resulting in a selective exclusion of target exon(s) is an approach that is already in clinical use to treat patients with Duchenne muscular dystrophy (DMD). Two currently approved therapies alter splicing to exclude exon 51 or exon 53 from the mature mRNA, restoring translation of a truncated, yet functionally active protein. However, the antisense PMO therapies allow to restore the wild-type (WT) DMD transcript and full-length dystrophin in patients with a single exon duplication.

Read More...

Leave A Reply