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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Détails bibliographiques
Publié dans:Mol Ther
Auteurs principaux: 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
Format: Artigo
Langue:Inglês
Publié: Nature Publishing Group 2016
Sujets:
Original Article
Accès en ligne: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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