Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

Advancement of RNAi-based therapeutics depends on effective delivery to the site of protein synthesis. Although intravenously administered, multi-component delivery vehicles have enabled small interfering RNA (siRNA) delivery and progression into clinical development, advances of single-component, s...

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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: Zimmermann, Tracy S., Karsten, Verena, Chan, Amy, Chiesa, Joseph, Boyce, Malcolm, Bettencourt, Brian R., Hutabarat, Renta, Nochur, Saraswathy, Vaishnaw, Akshay, Gollob, Jared
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363199/
https://ncbi.nlm.nih.gov/pubmed/28129130
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2016.10.019
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