Signs of progress in gene therapy for muscular dystrophy also warrant caution
- PMID: 22297820
- PMCID: PMC3277231
- DOI: 10.1038/mt.2011.307
Signs of progress in gene therapy for muscular dystrophy also warrant caution
Comment on
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Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.Mol Ther. 2012 Feb;20(2):456-61. doi: 10.1038/mt.2011.137. Epub 2011 Jul 19. Mol Ther. 2012. PMID: 21772257 Free PMC article.
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Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.Mol Ther. 2012 Feb;20(2):443-55. doi: 10.1038/mt.2011.237. Epub 2011 Nov 8. Mol Ther. 2012. PMID: 22068425 Free PMC article. Clinical Trial.
References
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- Monaco AP, Neve RL, Colletti-Feener C, Bertelson CJ, Kurnit DM., and, Kunkel LM. Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature. 1986;323:646–650. - PubMed
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- Koenig M, Monaco AP., and, Kunkel LM. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein. Cell. 1988;53:219–226. - PubMed
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