Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing

Delivery represents a significant barrier to the clinical advancement of oligonucleotide therapeutics for the treatment of neurological disorders, such as Huntington's disease. Small, endogenous vesicles known as exosomes have the potential to act as oligonucleotide delivery vehicles, but robus...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Didiot, Marie-Cécile, Hall, Lauren M, Coles, Andrew H, Haraszti, Reka A, Godinho, Bruno MDC, Chase, Kathryn, Sapp, Ellen, Ly, Socheata, Alterman, Julia F, Hassler, Matthew R, Echeverria, Dimas, Raj, Lakshmi, Morrissey, David V, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5112038/
https://ncbi.nlm.nih.gov/pubmed/27506293
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.126
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