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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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Detalles Bibliográficos
Autores principales: Beraza-Millor, Marina, Rodríguez-Castejón, Julen, Miranda, Jonatan, del Pozo-Rodríguez, Ana, Rodríguez-Gascón, Alicia, Solinís, María Ángeles
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10385692/
https://www.ncbi.nlm.nih.gov/pubmed/37514122
http://dx.doi.org/10.3390/pharmaceutics15071936