Challenges and Opportunities for Nucleic Acid Therapeutics

David R. Corey, Masad J. Damha, Muthiah Manoharan

Research output: Contribution to journalArticlepeer-review

21 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)8-13
Number of pages6
JournalNucleic Acid Therapeutics
Issue number1
StatePublished - Feb 2022


  • antisense
  • siRNA
  • therapeutic

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Drug Discovery


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