From the RNA world to the clinic.

Journal Article (Journal Article;Review)

The study of RNA has continually emphasized the structural and functional versatility of RNA molecules. This versatility has inspired translational and clinical researchers to explore the utility of RNA-based therapeutic agents for a wide variety of medical applications. Several RNA therapeutics, with diverse modes of action, are being evaluated in large late-stage clinical trials, and many more are in early clinical development. Hundreds of patients are enrolled in large trials testing messenger RNAs to combat cancer, small interfering RNAs to treat renal and hepatic disorders, and aptamers to combat ocular and cardiovascular disease. Results from these studies are generating considerable interest among the biomedical community and the public and will be important for the future development of this emerging class of therapeutic agents.

Full Text

Duke Authors

Cited Authors

  • Sullenger, BA; Nair, S

Published Date

  • June 17, 2016

Published In

Volume / Issue

  • 352 / 6292

Start / End Page

  • 1417 - 1420

PubMed ID

  • 27313039

Pubmed Central ID

  • PMC6035743

Electronic International Standard Serial Number (EISSN)

  • 1095-9203

Digital Object Identifier (DOI)

  • 10.1126/science.aad8709


  • eng

Conference Location

  • United States