Mice Cured of Autism

noahisaac writes "My brother just sent me an article he posted for the Rett Syndrome Research Foundation about a cure for Rett Syndrome, a form of autism. According to the article, researchers successfully re-introduced a fully functional version of the MECP2 gene into mice that had been born with damaged MECP2 genes. Contrary to their expectations, the mice improved. In the article's words, 'restoration of fully functional MECP2 over a four week period eradicated tremors and normalized breathing, mobility and gait in mice that had previously been fully symptomatic and, in some cases, only days away from death.' The ramifications for people suffering from Rett Syndrome are obvious, but mutations of the MECP2 gene are also believed to be the cause of 'classic' autism, and a number of other neurological disorders."

Comments

Runtime gene patching!

[jhantin]

So they spliced a stop codon into the middle of the relevant gene to disable it, then delivered an enzyme all the way to cell nuclei (!) to delete what they spliced in. The next step then it seems is then to find or engineer a proper enzyme to patch a naturally occurring gene defect -- they've basically proven that runtime patching of the genome works. Nice.

But adults may still be out of luck

[Michael Woodhams]

They've reversed (something like) Rett Syndrome in mice, showing that the nerve malfunctioning is reversable. In humans, however, missing vital developmental milestones is not reversable. E.g. normally we acquire grammar by age three, but if for some reason we don't acquire it before the age of about 10, we never will (or only very poorly.) So even if this treatment transfers to humans, it is unlikely to be a complete miracle cure for adult Rett Syndrome (or autistic) people.

Here's another article about it.