Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing

Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing

GM1 gangliosidosis (GM1) is a rare autosomal recessive neurogenerative lysosomal storage disease characterized by deficiency of beta-galactosidase (β-gal) and intralysosomal accumulation of GM1 ganglioside and other glycoconjugates. Resources for GM1 disease modelling are limited, and access to rele...

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Bibliographic Details
Main Authors: Allisandra K. Rha, Chloe L. Christensen, Shih-Hsin Kan, Jerry F. Harb, Perla Andrade-Heckman, Raymond Y. Wang
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Stem Cell Research
Subjects:
GM1 gangliosidosis
Lysosomal storage
Beta-galactosidase deficiency
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506124002502
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