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

• Hauptverfasser: Julen Rodríguez-Castejón (Verfasst von), Ana Alarcia-Lacalle (Verfasst von), Itziar Gómez-Aguado (Verfasst von), Mónica Vicente-Pascual (Verfasst von), María Ángeles Solinís Aspiazu (Verfasst von), Ana del Pozo-Rodríguez (Verfasst von), Alicia Rodríguez-Gascón (Verfasst von)
• Format: Buch
• Veröffentlicht: MDPI AG, 2021-05-01T00:00:00Z.
• Schlagworte: article
• Online-Zugang: 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...