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Adenovirus-mediated transfer of the acid alpha-glucosidase gene into fibroblasts, myoblasts and myotubes from patients with glycogen storage disease type II leads to high level expression of enzyme and corrects glycogen accumulation.
Nicolino MP, Puech JP, Kremer EJ, Reuser AJ, Mbebi C, Verdière-Sahuqué M, Kahn A, Poenaru L. Nicolino MP, et al. Among authors: poenaru l. Hum Mol Genet. 1998 Oct;7(11):1695-702. doi: 10.1093/hmg/7.11.1695. Hum Mol Genet. 1998. PMID: 9736771
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