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Generation of GLA-knockout human embryonic stem cell lines to model peripheral neuropathy in Fabry disease

Fabry disease is an X-linked glycolipid storage disorder caused by mutations in the GLA gene which result in a deficiency in the lysosomal enzyme alpha galactosidase A (AGA). As a result, the glycolipid substrate Gb3 accumulates in critical tissues and organs producing a progressive debilitating dis...

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Detalles Bibliográficos
Autores principales:	Kaneski, Christine R., Hanover, John A., Schueler Hoffman, Ulrike H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2022
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9449667/ https://www.ncbi.nlm.nih.gov/pubmed/36092250 http://dx.doi.org/10.1016/j.ymgmr.2022.100914

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