Lu, Qi-Long

UNCC

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POLYMER MEDIATED DELIVERY OF OLIGONUCLEOTIDE AND TRANS GENES TOWARD MORE EFFICIENT AND SPECIFIC THERAPIES FOR DYSTROPHINOPATHIES AND DYSTROGLYCANOPATHIES 2014 715 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...