Relieving Bottlenecks in RNA Drug Discovery for Retinal Diseases
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.
KeywordsRetinal degenerations Macular degenerations Gene therapy Posttranscriptional gene silencing Ribozyme Hammerhead ribozyme shRNA siRNA High throughput screening Breakthrough technology development
We thank the National Eye Institute (R01 EY13433, PI: Sullivan) (R24 EY016662, PI: M Slaughter), the Veterans Administration (Merit Grant 1I01BX000669-01), an Unrestricted grant from Research to Prevent Blindness, and a grant from the Oishei Foundation (Buffalo, NY).
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