| POLYMER MEDIATED DELIVERY OF OLIGONUCLEOTIDE AND TRANS GENES TOWARD MORE EFFICIENT AND SPECIFIC THERAPIES FOR DYSTROPHINOPATHIES AND DYSTROGLYCANOPATHIES |
2014 |
1038 |
Muscular dystrophies are genetic disorders caused by mutations in more than 30
genes, with no cure or effective treatment. Duchenne muscular dystrophy (DMD) is caused by frame-shift mutations in the dystrophin gene with little or no functional dystr... |