Gene Therapy Breakthrough For Autism Condition
Gene Therapy Breakthrough For Autism Condition
The British study of laboratory mice suggests a gene treatment can reverse the symptoms of Rett Syndrome, regarded as the most physically disabling of the autistic diseases.
The condition strikes mainly girls and leaves many in wheelchairs, suffering from tremors and breathing disorders.
The study, reported on-line by Science Express today, suggests that the mutant gene, MECP2, can successfully be replaced.
Healthy genes were administered to mice bred to be born with the Rett syndrome gene.
Researcher Professor Adrian Bird, of Edinburgh University, Scotland, said: "Like many other people, we expected that giving MeCP2 to mice that were already sick would not work.
"The idea that you could put back an essential component after the damage to the brain is done and recover an apparently normal mouse seemed farfetched, as nerve cells that developed in the absence of a key component were assumed to be irrevocably damaged.
"The results are gratifyingly clear, though, and must give hope to those who are affected by this distressing disorder."
The four week treatment eradicated tremors, restored breathing to normal and restored mobility and steady gait to the animals.
Monica Coenraads, a founder of the Rett Syndrome Research Foundation, said: "Dr. Bird’s astonishing results usher in a new era for Rett Syndrome and other autism spectrum disorders. The reversal experiments provide justification for aggressive exploration of next steps on all fronts, from drug discovery to gene correction."
And Professor Huda Zoghbi, who discovered the Rett syndrome gene, described the results as "extraordinary".
Professor Zoghbi, of Baylor College of Medicine, Texas, USA, said: "They are of relevance not only to Rett Syndrome but to a much broader class of disorders, including autism and schizophrenia.
"The successful restoration of normal function demonstrated in the mouse models suggests that if we can develop therapies to address the loss of MECP2 we may be able to reverse neurological damage in children and adults with Rett, autism and related neuropsychiatric disorders."
Last Updated: 09/02/2007 - 12:00 AM
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