Advances in oligonucleotide drug delivery
Name
Pages from ROBERTS-NRDD-2020-Advancesinoligonucleotidedrugdelivery-1.pdf
Description
Accepted version
Size
2.46 MB
Format
Adobe PDF
Checksum (MD5)
5c7052dbd9514ebb234d55d04208d94d
Author(s)
Roberts, Thomas C
Langer, Robert
Wood, Matthew JA
Date Issued
2020
Journal
Nature Reviews Drug Discovery
Publisher
Springer Science and Business Media LLC
Version
Author's final manuscript
Abstract
Oligonucleotides can be used to modulate gene expression via a range of processes including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation, non-coding RNA inhibition, gene activation and programmed gene editing. As such, these molecules have potential therapeutic applications for myriad indications, with several oligonucleotide drugs recently gaining approval. However, despite recent technological advances, achieving efficient oligonucleotide delivery, particularly to extrahepatic tissues, remains a major translational limitation. Here, we provide an overview of oligonucleotide-based drug platforms, focusing on key approaches — including chemical modification, bioconjugation and the use of nanocarriers — which aim to address the delivery challenge.
MIT Department
Massachusetts Institute of Technology. Department of Chemical Engineering
Koch Institute for Integrative Cancer Research at MIT
Terms of Use
Creative Commons Attribution-Noncommercial-Share Alike
Persistent DSpace Link
DOI of Published Version
https://doi.org/10.1038/S41573-020-0075-7
