Review 10.1172/JCI125228

Selective tissue targeting of synthetic nucleic acid drugs

Punit P. Seth, Michael Tanowitz, and C. Frank Bennett

Ionis Pharmaceuticals Inc., Carlsbad, California, USA.

Address correspondence to: C. Frank Bennett, Ionis Pharmaceuticals Inc., 2855 Gazelle Court, Carlsbad, California 92010, USA. Phone: 760.603.2336; Email: fbennett@ionisph.com.

Find articles by Seth, P. in: JCI | PubMed | Google Scholar |

Ionis Pharmaceuticals Inc., Carlsbad, California, USA.

Address correspondence to: C. Frank Bennett, Ionis Pharmaceuticals Inc., 2855 Gazelle Court, Carlsbad, California 92010, USA. Phone: 760.603.2336; Email: fbennett@ionisph.com.

Find articles by Tanowitz, M. in: JCI | PubMed | Google Scholar

Ionis Pharmaceuticals Inc., Carlsbad, California, USA.

Address correspondence to: C. Frank Bennett, Ionis Pharmaceuticals Inc., 2855 Gazelle Court, Carlsbad, California 92010, USA. Phone: 760.603.2336; Email: fbennett@ionisph.com.

Find articles by Bennett, C. in: JCI | PubMed | Google Scholar

First published January 28, 2019 - More info

Published in Volume 129, Issue 3 on March 1, 2019
J Clin Invest. 2019;129(3):915–925. https://doi.org/10.1172/JCI125228.
Copyright © 2019, American Society for Clinical Investigation
First published January 28, 2019 - Version history

Antisense oligonucleotides (ASOs) are chemically synthesized nucleic acid analogs designed to bind to RNA by Watson-Crick base pairing. Following binding to the targeted RNA, the ASO perturbs RNA function by promoting selective degradation of the targeted RNA, altering RNA intermediary metabolism, or disrupting function of the RNA. Most antisense drugs are chemically modified to enhance their pharmacological properties and for passive targeting of the tissues of therapeutic interest. Recent advances in selective tissue targeting have resulted in a newer generation of ASO drugs that are more potent and better tolerated than previous generations, spawning renewed interest in identifying selective ligands that enhance targeted delivery of ASOs to tissues.

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