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Usage Information

Selective tissue targeting of synthetic nucleic acid drugs
Punit P. Seth, … , Michael Tanowitz, C. Frank Bennett
Punit P. Seth, … , Michael Tanowitz, C. Frank Bennett
Published January 28, 2019
Citation Information: J Clin Invest. 2019;129(3):915-925. https://doi.org/10.1172/JCI125228.
View: Text | PDF
Review

Selective tissue targeting of synthetic nucleic acid drugs

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Abstract

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.

Authors

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

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Usage data is cumulative from May 2024 through May 2025.

Usage JCI PMC
Text version 1,205 482
PDF 172 82
Figure 235 4
Table 199 0
Citation downloads 80 0
Totals 1,891 568
Total Views 2,459
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