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Generation of an in vitro model for peripheral neuropathy in Fabry disease using CRISPR-Cas9 in the nociceptive dorsal root ganglion cell line 50B11

Fabry disease is a glycosphingolipid storage disorder that is caused by a genetic deficiency of the lysosomal enzyme alpha-galactosidase A (AGA, EC 3.2.1.22). As a result, the glycolipid substrate, globotriaosylceramide (Gb3) accumulates in various cell types throughout the body producing a multisys...

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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/PMC9248215/ https://www.ncbi.nlm.nih.gov/pubmed/35782611 http://dx.doi.org/10.1016/j.ymgmr.2022.100871

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