Gene therapy for muscular dystrophy: Lessons learned and path forward

► Molecular therapeutic approaches to treat muscular dystrophies are discussed. ► Exon skipping targets pre-mRNA allowing one or more exons to be omitted. ► Proof of principle for readthrough of stop codons has been established in DMD. ► Mini-dystrophin gene transfer resulted in an immune response,...

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Veröffentlicht in:	Neuroscience letters 2012-10, Vol.527 (2), p.90-99
Hauptverfasser:	Mendell, Jerry R., Rodino-Klapac, Louise, Sahenk, Zarife, Malik, Vinod, Kaspar, Brian K., Walker, Christopher M., Clark, K. Reed
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Alpha-sarcoglycan Clinical Trials as Topic Codon, Terminator Creatine Kinase - genetics Dependovirus - genetics Dystrophin Dystrophin - genetics Dystrophin - metabolism Exon skipping Exons Follistatin Follistatin - genetics Genetic Therapy - methods Genetic Vectors Humans Indexing in process Muscular Dystrophies - genetics Muscular Dystrophies - metabolism Muscular Dystrophies - therapy Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - metabolism Muscular Dystrophy, Duchenne - therapy Mutation Mutation suppression Myositis, Inclusion Body - genetics Myositis, Inclusion Body - metabolism Myositis, Inclusion Body - therapy Oxadiazoles - therapeutic use Promoter Regions, Genetic RNA Precursors - genetics Sarcoglycans - genetics Sarcoglycans - metabolism
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