A4GALT-targeting siRNA lipid nanoparticles ameliorate Fabry disease phenotype: Greater efficacy in endothelial cells than in podocytes

A4GALT-targeting siRNA lipid nanoparticles ameliorate Fabry disease phenotype: Greater efficacy in endothelial cells than in podocytes

In this study, we explore the therapeutic feasibility of globotriaosylceramide (Gb3) synthase (A4GALT)-specific siRNA-loaded polyhistidine (pHis)-incorporated lipid nanoparticles (HLNPs) for Fabry disease (FD). HLNPs were developed to deliver siRNAs targeting A4GALT using a microfluidic device, with...

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Main Authors: Yoo Jin Shin, Hanbi Lee, Xianying Fang, Sheng Cui, Sun Woo Lim, Kang In Lee, Jae Young Lee, Hong Lim Kim, Yuna Oh, Can Li, Chul Woo Yang, Gayeon You, Hyeondo Lee, Hyejung Mok, Byung Ha Chung
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
Fabry disease
A4GALT siRNA
polyhistidine-incorporating lipid nanoparticle
human-induced pluripotent-stem-cell-derived endothelial cell
podocyte
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125001271
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