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. 2022 Feb;32(1):8-13.
doi: 10.1089/nat.2021.0085. Epub 2021 Dec 17.

Challenges and Opportunities for Nucleic Acid Therapeutics

David R Corey  1 , Masad J Damha  2 , Muthiah Manoharan  3
Affiliations

Challenges and Opportunities for Nucleic Acid Therapeutics

David R Corey et al. Nucleic Acid Ther. 2022 Feb.

Abstract

After decades overcoming difficult problems, antisense oligonucleotide (ASO), duplex RNA (siRNA), and messenger RNA (mRNA) nucleic acid therapeutic strategies are finally demonstrating clinical benefits. This success presents new challenges. What goals remain for basic research? Will there be an explosion of clinical applications that benefit many patients with different diseases, or will success be restricted to diseases that are ideal for the application of current technologies? The aim of this perspective is to describe a selection of the major goals for the next decade.

Keywords: antisense; siRNA; therapeutic.

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Conflict of interest statement

M. Manoharan is employed by Alnylam Pharmaceuticals. M.J. Damha is a member of the Scientific Advisory Board of Aro Biotherapeutics, AUM LifeTech, and Deep Genomics.

Figures

FIG. 1.
FIG. 1.
Chemical modifications involved in approved nucleic acids medicines.
See this image and copyright information in PMC

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