Use of the dog model for Duchenne muscular dystrophy in gene therapy trials.

@article{Howell1997UseOT,
  title={Use of the dog model for Duchenne muscular dystrophy in gene therapy trials.},
  author={James M. Howell and Susan Fletcher and Byron A. Kakulas and Maureen O'hara and Hanns Lochmuller and George Karpati},
  journal={Neuromuscular disorders : NMD},
  year={1997},
  volume={7 5},
  pages={325-8}
}
Golden retriever muscular dystrophy (GRMD) is an excellent model for the study of the efficacy of gene therapy in dystrophin deficient myopathies for there are many similarities between affected dogs and Duchenne muscular dystrophy (DMD) in boys. GRMD is not caused by deletion mutation but results from a point mutation in the consensus splice acceptor in intron 6 of the canine dystrophin gene. As a result exon 7 is skipped during processing of the GRMD dystrophin messenger RNA. We have… CONTINUE READING