DMD CLINICAL THERAPIES II

0

Duchenne muscular dystrophy (DMD) is a fatal childhood disease caused by mutations in the DMD gene that abolish the expression of dystrophin protein. Golden retriever muscular dystrophy (GRMD) is a naturally occurring, genetically homologous DMD model that closely recapitulates the progressive and variable phenotype seen in patients. A point mutation in the intron 6 DMD acceptor splice site in affected dogs disrupts the DMD reading frame, leading to exon 7 skipping, a stop codon in exon 8, and subsequent loss of dystrophin protein.

Read More...

Leave A Reply