Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach

Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach

More than two thirds of Lysosomal Storage Diseases (LSDs) present central nervous system involvement. Nevertheless, only one of the currently approved therapies has an impact on neuropathology. Therefore, alternative approaches are under development, either addressing the underlying enzymatic defect...

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Bibliographic Details
Main Authors: Maria Francisca Coutinho, Juliana Inês Santos, Liliana S. Mendonça, Liliana Matos, Maria João Prata, Amália S. Jurado, Maria C. Pedroso de Lima, Sandra Alves
Format: Article
Language:English
Published: MDPI AG 2020-08-01
Series:International Journal of Molecular Sciences
Subjects:
lysosomal storage diseases (LSDs)
neuropathy
substrate reduction therapy (SRT)
RNA interference (RNAi)
siRNA nanodelivery systems
stable nucleic acid lipid particles (SNALPs)
Online Access:https://www.mdpi.com/1422-0067/21/16/5732

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https://www.mdpi.com/1422-0067/21/16/5732

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