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Nanomedicine and Nanoscale Delivery (Focus Group - NND)

(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.

Learning Objectives:

  • Developing novel therapies for lysosomal storage disorders based on target delivery of enzyme.
Daniel Cerri – Level 5-Technical Training Fellowship funded by São Paulo Research Foundation – FAPESP, School of Pharmaceutical Sciences of Ribeirao Preto, University of São Paulo; Lívia Depieri – Level 5-Technical Training Fellowship funded by São Paulo Research Foundation – FAPESP, School of Pharmaceutical Sciences of Ribeirao Preto, University of São Paulo; Maria Vitória Bentley – Professor Doctor, School of Pharmaceutical Sciences of Ribeirao Preto, University of São Paulo