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Updated: May 13, 2026
Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
Recent progress in oligonucleotide therapeutics: antisense to aptamers
1The University of Toledo, Department of Biological Sciences, Toledo, OH 43606, USA +1 419 530 1555 ; +1 419 530 7737 ; dleaman@utnet.utoledo.edu.
Background:
Antisense and other RNA-targeting strategies have the potential to impact a broad array of human diseases. Although the methodological approaches for achieving targeted RNA degradation have changed over the past 5 - 10 years, the technological hurdles facing these and other nucleic acid-based drugs have remained fairly constant.
Objective:
To provide an update on the clinical status of several antisense compounds and discuss methodological strategies for improving efficacy of antisense compounds and other nucleic acid therapeutics.
Method:
Some of the clinical advances in antisense therapeutics, including a description of sugar or backbone modifications and delivery approaches for improving antisense efficacy are highlighted; a few alternative nucleic acid strategies are discussed.
Conclusion:
Although a variety of technological issues continue to hamper antisense progress towards the clinic, advances in stabilization and delivery have provided new hope for using nucleic acids as drugs.
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