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α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice

Fabry disease (FD) is a monogenic X-linked lysosomal storage disorder caused by a deficiency in the lysosomal enzyme α-Galactosidase A (α-Gal A). It is a good candidate to be treated with gene therapy, in which moderately low levels of enzyme activity should be sufficient for clinical efficacy. In t...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Pharmaceutics
Prif Awduron: Rodríguez-Castejón, Julen, Alarcia-Lacalle, Ana, Gómez-Aguado, Itziar, Vicente-Pascual, Mónica, Solinís Aspiazu, María Ángeles, del Pozo-Rodríguez, Ana, Rodríguez-Gascón, Alicia
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: MDPI 2021
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC8224287/
https://ncbi.nlm.nih.gov/pubmed/34064206
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/pharmaceutics13060771
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