Novel Golden Lipid Nanoparticles with Small Interference Ribonucleic Acid for Substrate Reduction Therapy in Fabry Disease

Novel Golden Lipid Nanoparticles with Small Interference Ribonucleic Acid for Substrate Reduction Therapy in Fabry Disease

Substrate reduction therapy (SRT) has been proposed as a new gene therapy for Fabry disease (FD) to prevent the formation of globotriaosylceramide (Gb3). Nanomedicines containing different siRNA targeted to Gb3 synthase (Gb3S) were designed. Formulation factors, such as the composition, solid lipid...

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Bibliographic Details
Main Authors: Marina Beraza-Millor, Julen Rodríguez-Castejón, Jonatan Miranda, Ana del Pozo-Rodríguez, Alicia Rodríguez-Gascón, María Ángeles Solinís
Format: Article
Language:English
Published: MDPI AG 2023-07-01
Series:Pharmaceutics
Subjects:
substrate reduction therapy
solid lipid nanoparticles
siRNA
Fabry disease
Gb3 synthase
gold nanoparticles
Online Access:https://www.mdpi.com/1999-4923/15/7/1936

Internet

https://www.mdpi.com/1999-4923/15/7/1936

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