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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Detalles Bibliográficos
Autores principales: Rodríguez-Castejón, Julen, Alarcia-Lacalle, Ana, Gómez-Aguado, Itziar, Vicente-Pascual, Mónica, Solinís Aspiazu, María Ángeles, del Pozo-Rodríguez, Ana, Rodríguez-Gascón, Alicia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8224287/
https://www.ncbi.nlm.nih.gov/pubmed/34064206
http://dx.doi.org/10.3390/pharmaceutics13060771

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8224287/
https://www.ncbi.nlm.nih.gov/pubmed/34064206
http://dx.doi.org/10.3390/pharmaceutics13060771

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