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

• Main Authors: Julen Rodríguez-Castejón (Author), Ana Alarcia-Lacalle (Author), Itziar Gómez-Aguado (Author), Mónica Vicente-Pascual (Author), María Ángeles Solinís Aspiazu (Author), Ana del Pozo-Rodríguez (Author), Alicia Rodríguez-Gascón (Author)
• Format: Book
• Published: MDPI AG, 2021-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...