AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

GM1 gangliosidosis is a rare, inherited neurodegenerative disorder caused by mutations in the GLB1 gene, which encodes the lysosomal hydrolase acid β-galactosidase (β-gal). β-gal deficiency leads to toxic accumulation of GM1 ganglioside, predominantly in the central nervous system (CNS), resulting i...

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Bibliographic Details

Main Authors:	Michaël Hocquemiller, Laura Giersch, Xin Mei, Amanda L. Gross, Ashley N. Randle, Heather L. Gray-Edwards, Judith A. Hudson, Sophia Todeasa, Lorelei Stoica, Douglas R. Martin, Miguel Sena-Esteves, Karen Aiach, Ralph Laufer
Format:	Artigo
Language:	Inglês
Published:	Elsevier 2022-12-01
Series:	Molecular Therapy: Methods & Clinical Development
Subjects:	AAV gangliosidosis GM1 gene therapy lysosomal storage disease
Online Access:	http://www.sciencedirect.com/science/article/pii/S2329050122001462
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