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Relieving bottlenecks in RNA drug discovery for retinal diseases

  • Jack M. Sullivan
  • , Edwin H. Yau
  • , R. Thomas Taggart
  • , Mark C. Butler
  • , Tiffany A. Kolniak
  • SUNY Buffalo

Research output: Chapter in Book/Report/Conference proceedingConference contributionpeer-review

8 Scopus citations

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 to efficiently and rapidly assess a set of biological and experimental variables engaged in RNA drug discovery.

Original languageEnglish
Title of host publicationRetinal Degenerative Diseases
EditorsMatthew LaVail, Robert Anderson, Christian Grimm, John Ash, Joe Hollyfield
Pages145-153
Number of pages9
DOIs
StatePublished - 2012

Publication series

NameAdvances in Experimental Medicine and Biology
Volume723
ISSN (Print)0065-2598

Keywords

  • Breakthrough Technology Development
  • Gene Therapy
  • Hammerhead Ribozyme
  • High Throughput Screening
  • Macular Degenerations
  • Posttranscriptional Gene Silencing
  • Retinal Degenerations
  • Ribozyme
  • shRNA
  • siRNA

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