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In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion

Globoid Cell Leukodystrophy (GLD) is a lysosomal storage disease (LSD) caused by inherited defects of the β-galactosylceramidase (GALC) gene. The infantile forms display a rapid and aggressive central and peripheral nervous system (CNS and PNS) dysfunction. No treatments are available for GLD patien...

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Detalles Bibliográficos
Autores principales: Ricca, Alessandra, Cascino, Federica, Morena, Francesco, Martino, Sabata, Gritti, Angela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7396597/
https://www.ncbi.nlm.nih.gov/pubmed/32850960
http://dx.doi.org/10.3389/fmolb.2020.00167