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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Vydáno v:Int J Mol Sci
Hlavní autoři: Coutinho, Maria Francisca, Santos, Juliana Inês, S. Mendonça, Liliana, Matos, Liliana, Prata, Maria João, S. Jurado, Amália, Pedroso de Lima, Maria C., Alves, Sandra
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2020
Témata:
Review
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7461213/
https://ncbi.nlm.nih.gov/pubmed/32785133
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21165732
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