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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Autors principals:	Alessandra Ricca, Federica Cascino, Francesco Morena, Sabata Martino, Angela Gritti
Format:	Artigo
Idioma:	Inglês
Publicat:	Frontiers Media S.A. 2020-07-01
Col·lecció:	Frontiers in Molecular Biosciences
Matèries:	globoid cell leukodystrophy lentiviral vectors chimeric GALC enzyme neural stem/progenitor cells hematopoietic stem/progenitor cells gene therapy
Accés en línia:	https://www.frontiersin.org/article/10.3389/fmolb.2020.00167/full
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