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 an RNA copy, which then exits the nucleus and enters the ribosome in the cytoplasm where proteins are manufactured.
Occasionally, however, the RNA has a mistake in it and it doesn’t match the DNA template. 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. This approach will require a unique therapeutic designed for each point mutation.
What it is
DNA is the genetic instructions to make an organism. In order to access the instructions, they must first be copied to RNA and then translated to protein. During the copying phase errors sometimes happen. The body has an innate editing mechanism that detects and corrects these errors. This naturally occurring mechanism is now being harnessed to correct MECP2 mutations.
Why it matters
RNA editing is an important alternative to DNA editing which can cause unwanted mutations (‘off-target effects). Because unused RNAs are quickly degraded, any errors introduced by a therapeutic would be washed out, rather than staying with a person forever. RNA editing may also be less likely to cause an immune reaction as the editing mechanism is naturally found in humans. Likewise RNA editing might provide a more natural response than introducing an external, engineered gene. A disadvantage is that currently RNA editing can only fix A to G mutations. Scientists are looking for ways to engineer new enzymes that could expand RNA-editing capabilities.
Researchers have successfully edited and corrected the RNA in brain cells in Rett mice to successfully generate normal MECP2 protein at levels that are very encouraging. RSRT is funding scientists at the highest caliber who are consistently making breakthroughs in the RNA editing field. Importantly, a number of biopharmas are now pursuing programs in Rett Syndrome.
There would be no Vico Therapeutics had RSRT not funded my lab.
A cure for Rett.
You want it.
We want it.
Great achievements happen when people stand up and say I’m in. I will be part of the solution!
Rett may be a rare disorder but together we are powerful.