(330) Immunoliposomes as a targeted delivery system for enzyme replacement therapy in Fabry disease

Introduction: Fabry disease is a lysosomal storage disease that affect mainly the cardiac tissue (1). It causes the storage of glycolipids, such as globotriaosylceramide, in lysosomes, due to a default in the expression of the enzyme α-galactosidase A (GLA) (2). This work proposes the delivery of the GLA to cardiomyocytes by immunoliposomes functionalized with PEG and a monoclonal antibody to integrin alpha-7 (ITGA-7), a receptor highly expressed in these cells (3).

(1) Azevedo, et al., 2021.
(2) Cabrera, et al., 2016.
(3) Yuasa, et al., 2017.