New Molecular Tools for Directed Editing of MeCP2 Mutations Associated with Rett Syndrome
Peter Beal, PhD | UC Davis
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 it and the bases don’t match up exactly. Nature has come up with a solution to address 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.
Peter Beal’s project hijacks this naturally occurring process and puts it to work to fix mutations in the MeCP2 RNA. This particular approach would eventually lead to an intervention that requires dosing an individual with Rett on a regular basis. The advantage of multiple dosing is that it allows for titration of the dose to individually optimize benefit in each person. This permits timing of the treatment to be tailored to each patient in a precise way.
Peter Beal is a world expert in RNA editing and understanding of ADAR biology. He is collaborating with the lab of Gail Mandel who has been studying MECP2 for many years.