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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Detalles Bibliográficos
Autores principales: Coutinho, Maria Francisca, Santos, Juliana Inês, S. Mendonça, Liliana, Matos, Liliana, Prata, Maria João, S. Jurado, Amália, Pedroso de Lima, Maria C., Alves, Sandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7461213/
https://www.ncbi.nlm.nih.gov/pubmed/32785133
http://dx.doi.org/10.3390/ijms21165732

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7461213/
https://www.ncbi.nlm.nih.gov/pubmed/32785133
http://dx.doi.org/10.3390/ijms21165732

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