Can gene therapy cure autism ? and cancer? | Health Blog

Purkinje nerve cell (David McCarthy/Science Photo Library)

A fascinating new?Harvard Medical School study points to a single gene as a cause of autism-like behavior, with rapamycin, a commonly used immunosuppresant drug, working effectively to prevent these symptoms. It?s the first time, they say, that researchers have identified a molecular component for the cerebellum?s role in autism.

The research looks closely at how Purkinje cells, which are essential for cerebellar function, are fewer in number in patients with autism. Mice who have copies of the TSC1 gene in their Purkinje cells deleted, exhibit typical symptoms of autism, including abnormal social intereractions, repetitive behaviors and abnormal communication.

This is right on the heels of an equally fascinating New York Times story, In Treatment for Leukemia, Glimpses for the Future, in which a cancer scientist, Dr. Lukas Wartman, experienced remission from adult acute lymphoblastic leukemia after researchers identified one of his genes that was in overdrive, generating huge amounts of protein spurring his cancer?s growth, and then tried out a new drug aimed at shutting down the malfunctioning gene.

It could be a whole new way of looking at and curing what had once seemed incurable ? if scientists find a way to make genome sequencing more generally available and affordable, paid for by insurance ? which the New York Times speculates could take anywhere from a few years to a decade ? and drug companies and biotechs invest in research to attack faulty genes rather than tumors.

Source: http://healthblog.dallasnews.com/2012/07/can-gene-therapy-cure-autism-and-cancer.html/

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