Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation

Title
Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation
Authors
Keywords
induced pluripotent stem cells, lysosomal storage disease, GM1 gangliosidosis, drug development
Journal
Stem Cell Reports
Volume 14, Issue 5, Pages 909-923
Publisher
Elsevier BV
Online
2020-04-16
DOI
10.1016/j.stemcr.2020.03.012

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