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Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatment except palliative therapy. There are several promising genetic approaches, including viral delivery of the missing dystrophin gene, read-through of translation stop codons, exon skipping to restore the reading frame and increased expression of the compensatory utrophin gene. The lessons learned from these approaches will be applicable to many other disorders.

Original publication

DOI

10.1038/nrg3460

Type

Journal article

Journal

Nat Rev Genet

Publication Date

06/2013

Volume

14

Pages

373 - 378

Keywords

Animals, Clinical Trials as Topic, Dependovirus, Dystrophin, Gene Expression, Genetic Therapy, Genetic Vectors, Humans, Muscular Dystrophy, Duchenne, Mutation, Protein Structure, Tertiary, Utrophin