Recent Progress in Oligonucleotide Therapeutics: Antisense to Aptamers
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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.
Leaman, D. W.
(2008). Recent Progress in Oligonucleotide Therapeutics: Antisense to Aptamers. Expert Opinion on Drug Discovery, 3 (9), 997-1009.