RT Journal
A1 Kuehn BM
T1 MUscular dystrophy patch
JF JAMA
JO JAMA
YR 2009
FD May 6
VO 301
IS 17
SP 1758
OP 1758
DO 10.1001/jama.2009.569
UL http://dx.doi.org/10.1001/jama.2009.569
AB 
A variety of frameshift or nonsense mutations in the DMD gene interfere with the production of the protein dystrophin and cause progressive muscle weakness in patients with DMD. Previous studies have demonstrated that an intravenous exon-skipping drug, which covers up such mutations, could help rescue dystrophin production in mice with a DMD-like condition; intramuscular injections also have been shown to locally restore dystrophin production in 4 patients.