TY  - JOUR
T1  - MUscular dystrophy patch
AU  - Kuehn BM
Y1  - 2009/05/06
N1  - 10.1001/jama.2009.569
JO  - JAMA
SP  - 1758
EP  - 1758
VL  - 301
IS  - 17
N2  - 
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.

SN  - 0098-7484
M3  - doi: 10.1001/jama.2009.569
UR  - http://dx.doi.org/10.1001/jama.2009.569
ER  -