New Editing Enzymes for RNA

Gail Mandel, PhD | John Sinnamon, PhD

$345,000 AWARDED

DNA is made up of bases that pair together, A, T, C and G to make the double helix we are all familiar with.  This helix unzips to make RNA, which then exits the nucleus and enters the ribosome where proteins are manufactured.  The bases in the DNA and RNA should be exactly the same.

Occasionally however the RNA has a mistake in and the bases don’t match up exactly.  Nature has come up with a solution for this kind of mistake. All of us have an enzyme in our cells called ADAR that acts as an editor, looking for and correcting mistakes in RNA. This process is called RNA editing.

Gail Mandel and lab member, John Sinnamon, are employing RNA editing by introducing their own version of ADARs, which could be considered super editors with increased specificity.  Ultimately this therapeutic approach would be administered as a one-time treatment similarly to a gene therapy product.

The Mandel lab has already successfully edited and corrected the RNA  in brain cells in Rett mice to successfully generate normal MeCP2 protein. They are now administering the RNA editing treatment to Rett mice to see if their symptoms are alleviated.

The Mandel lab has been focused on Rett Syndrome for a number of years and has an extensive track record of important discoveries. For this project they are collaborating with Peter Beal of UCSD.



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