Relieving bottlenecks in RNA drug discovery for retinal diseases

Adv Exp Med Biol. 2012:723:145-53. doi: 10.1007/978-1-4614-0631-0_20.

Abstract

The development of efficacious and safe post transcriptional gene silencing (PTGS) agents is a challenging scientific endeavor that embraces “biocomplexity” at many levels. The target mRNA exhibits a level of structural complexity that profoundly limits annealing of PTGS agents. PTGS agents are macromolecular RNAs that must be designed to fold into catalytically active structures able to cleave the target mRNA. Pushing into and beyond the biological complexity requires new technologies for high throughput screening (HTS) to efficiently and rapidly assess a set of biological and experimental variables engaged in RNA Drug Discovery (RDD).

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods*
  • Drug Discovery / trends
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • RNA Interference*
  • RNA, Catalytic / chemistry
  • RNA, Catalytic / genetics
  • Retinal Degeneration / drug therapy*
  • Retinal Degeneration / genetics*
  • Thionucleotides / chemistry
  • Thionucleotides / genetics

Substances

  • RNA, Catalytic
  • Thionucleotides
  • hammerhead ribozyme
  • fomivirsen