Strand bias in oligonucleotide-mediated dystrophin gene editing.

Abstract excerpt

Defects in the dystrophin gene cause the severe degenerative muscle disorder, Duchenne muscular dystrophy (DMD). Among the gene therapy approaches to DMD under investigation, a gene editing approach using oligonucleotide vectors has yielded encouraging results. Here, we extend our studies of gene editing with self-pairing, chimeric RNA/DNA oligonucleotides (RDOs) to the use of oligodeoxynucleotides (ODNs) to...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Dystrophin
• Share to Gene Transfer Techniques
• Share to In Situ Hybridization, Fluorescence