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Nucleic Acid Ther. 2018 Jun;28(3):128-136. doi: 10.1089/nat.2018.0725. Epub 2018 May 10.

Improving siRNA Delivery In Vivo Through Lipid Conjugation.

Author information

1
1 RNA Therapeutics Institute, University of Massachusetts Medical School , Worcester, Massachusetts.
2
2 Department of Molecular Medicine, University of Massachusetts Medical School , Worcester, Massachusetts.

Abstract

RNA interference (RNAi)-based therapeutics are approaching clinical approval for genetically defined diseases. Current clinical success is a result of significant innovations in the development of chemical architectures that support sustained, multi-month efficacy in vivo following a single administration. Conjugate-mediated delivery has established itself as the most promising platform for safe and targeted small interfering RNA (siRNA) delivery. Lipophilic conjugates represent a major class of modifications that improve siRNA pharmacokinetics and enable efficacy in a broad range of tissues. Here, we review current literature and define key features and limitations of this approach for in vivo modulation of gene expression.

KEYWORDS:

delivery; lipid conjugation; siRNA

PMID:
29746209
PMCID:
PMC5994667
DOI:
10.1089/nat.2018.0725
[Indexed for MEDLINE]
Free PMC Article

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