Mice with a gene mutation related to Rett syndrome breathe more easily and live longer after an RNA-editing treatment, according to a new study. The treatment partially restores expression of the mutated gene in the brainstem, which controls such basic functions as respiration.
The method could potentially be tailored for use in people with Rett syndrome, says lead investigator Gail Mandel, professor of biochemistry and molecular biology at Oregon Health & Science University in Portland. The mice have a mutation in the MECP2 gene that involves a single DNA letter swap from guanosine (G) to adenosine (A), which mirrors a mutation seen in people.