Reversing Pathology in an Aggravated Fabry Mouse Model Using Low-Dose Engineered Human Alpha-Galactosidase A AAV Gene Therapy

Reversing Pathology in an Aggravated Fabry Mouse Model Using Low-Dose Engineered Human Alpha-Galactosidase A AAV Gene Therapy

<b>Background/Objectives</b>: Fabry disease is an X-linked disorder caused by lysosomal accumulation of glycosphingolipids due to the deficiency of α-Galactosidase (α-GAL), which leads to pathology in multiple organ systems. The standard of care is enzyme replacement therapy (ERT) with r...

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Bibliographic Details
Main Authors: Wanida Ruangsiriluk, Mugdha Deshpande, Natalia Boukharov, Girija Rajarshi, Shreya Mukherji, Shipeng Yuan, Jennifer Wiseman, Nancy Chen, Eric Park, Hyelim Cho, Rizwana Islam
Format: Article
Language:English
Published: MDPI AG 2025-02-01
Series:Biomedicines
Subjects:
Fabry disease
lysosomal storage disease
adeno-associated virus (AAV)
rAAV9
AAV gene therapy
alpha-Galactosidase A (<i>GLA</i>)
Online Access:https://www.mdpi.com/2227-9059/13/3/577
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