Genetic correction strategies for Duchenne Muscular Dystrophy and their impact on the heart.

ABSTRACT:

Background

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder with early childhood onset characterized by profound loss of muscle strength and associated cardiomyopathy. DMD affects is most often caused by deletions involving single or multiple exons that disrupt the open reading frame of the DMD gene. Mutations causing loss or premature truncation of dystrophin result in dystrophin protein deficiency, which renders the plasma membrane of skeletal myofibers and cardiomyocytes weakened.

Aim of review

Genetic correction is in use to treat DMD, since several drugs have been already approved which partially restore dystrophin production through the use of antisense oligonucleotides. There are multiple ongoing clinical trials to evaluate the efficacy of tr

SUBMITTER: Johnston JR 

PROVIDER: S-EPMC8656413 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature