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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Bibliografske podrobnosti
izdano v: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
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2016
Teme:
Original Article
Online dostop: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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