RNA Molecule for Precisely Regulating Gene Expression

Technology #14710

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Chen Ling
Arun Srivastava
Managed By
April Kilburn
Assistant Director 352-392-8929
Patent Protection
US Patent Pending US2016/0102321
PCT Patent Application WO2014/186746

Boosts or Represses Gene Expression to Treat Diseases in Humans and Animals

This hairpin RNA molecule locates at the end of mRNA in the cell and controls its translation into proteins, enhancing control over gene expression when compared to available siRNA technologies. Controlling gene expression is an important aspect of gene therapy. Absent or abnormally functioning genes can be replaced, altered, or supplemented to treat disease and improve patient health. Existing technologies are only capable of repressing gene expression. Researchers at the University of Florida have developed an RNA molecule that can also increase gene expression levels. Animal testing has produced positive results. This RNA molecule could have many potential applications in gene therapy and other types of genomics research. The global gene therapy market is expected to reach $794 million by 2017.


A hairpin-shaped RNA molecule for controlling gene regulation and treating diseases using gene therapy, which has proven successful in animals


  • Boosts or represses gene expression, making it preferable to existing technologies that can only repress gene expression
  • Complements existing technologies, creating a wider portfolio of tools for researchers
  • Enables greater precision in regulating gene expression levels, providing more flexibility for research and treatment


Researchers have discovered an RNA molecule that affects gene expression rates. The RNA molecule locates at the end of the mRNA strand that will be translated into a protein. This prevents translation from occurring and lowers expression rates. Adding other proteins can modify the behavior of the mRNA to increase expression levels.