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Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle.
Mol Ther. 2007 Feb;15(2):320-9. doi: 10.1038/sj.mt.6300027.
Mol Ther. 2007.
PMID: 17235310
Free article.
Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy.
Sun B, Young SP, Li P, Di C, Brown T, Salva MZ, Li S, Bird A, Yan Z, Auten R, Hauschka SD, Koeberl DD.
Sun B, et al. Among authors: salva mz.
Mol Ther. 2008 Aug;16(8):1366-71. doi: 10.1038/mt.2008.133. Epub 2008 Jun 17.
Mol Ther. 2008.
PMID: 18560415
Free PMC article.
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