Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan
Weismann, Cara M, Ferreira, Jennifer, Keeler, Allison M, Su, Qin, Qui, Linghua, Shaffer, Scott A, Xu, Zuoshang, Gao, Guangping, Sena-Esteves, Miguel
Published in Human molecular genetics (01.08.2015)
Published in Human molecular genetics (01.08.2015)
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