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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Dades bibliogràfiques
Publicat a:Int J Mol Sci
Autors principals: Coutinho, Maria Francisca, Santos, Juliana Inês, S. Mendonça, Liliana, Matos, Liliana, Prata, Maria João, S. Jurado, Amália, Pedroso de Lima, Maria C., Alves, Sandra
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2020
Matèries:
Review
Accés en línia: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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