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Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.

Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G, Sena-Esteves M. Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan. Hum Mol Genet. 2015 Aug 01; 24(15):4353-64.

View in: PubMed