Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics(clinical),Genetics,Molecular Biology
Link
http://www.nature.com/articles/nrg3460.pdf
Reference50 articles.
1. Emery, A. E. H. Muscular Dystrophy (The Facts) (Oxford Univ. Press, 2008).
2. Cohn, R. D. & Campbell, K. P. Molecular basis of muscular dystrophies. Muscle Nerve 23, 1456–1471 (2000).
3. Aartsma-Rus, A. et al. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum. Mutat. 30, 293–299 (2009).
4. Fairclough, R. J., Perkins, K. J. & Davies, K. E. Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy. Curr. Gene Ther. 12, 206–244 (2012).
5. Neri, M. et al. Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human. Neuromuscul. Disord. 17, 913–918 (2007).
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