Enzyme-Loaded Immunoliposomes Targeting Cardiomyocytes as a Therapeutic Strategy for Fabry Disease

Luiz Souza – PhD student, School of Pharmaceutical Sciences of Ribeirao Preto, University of Sao Paulo; Daniel Cerri – Postdoctoral, School of Pharmaceutical Sciences of Ribeirao Preto, University of Sao Paulo; Livia Depieri – Postdoctoral, School of Pharmaceutical Sciences of Ribeirao Preto, University of Sao Paulo

Introduction:

Fabry disease (FD) is a lysosomal storage disorder caused by α-galactosidase A (GLA) deficiency, leading to globotriaosylceramide (Gb3) accumulation, and cardiac involvement is the primary cause of mortality [1]. Current Enzyme Replacement Therapy (ERT) is limited by short half-life and poor uptake in cardiac tissue [2]. This project proposes a targeted delivery system using immunoliposomes. By microfluidics and functionalizing surfaces with anti-integrin α7 antibodies, we enhanced GLA delivery specifically to cardiomyocytes, offering a strategy to mitigate FD-associated cardiomyopathy.