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UCI research turns the corner on autism
Date:1/15/2013

ting impact on autism worldwide because of our focus on how molecular, cellular and brain functions are altered and how drugs affect each level. We are confident that the program and its goals are feasible because all of the necessary pieces are already in place at UC Irvine."

The center's role is particularly important today, he says, because major pharmaceutical companies have mostly neglected autism, and breakthroughs in decoding the human genome are now providing researchers an unprecedented opportunity to understand the innermost workings of autism.

More than a decade ago, it was discovered that cancer is a genetic disease, making the defective genes a target of drug discovery campaigns and resulting in the first round of cancer drugs specific to the molecular lesion. Thus began an optimistic new era of cancer treatments.

Gargus thinks autism research is at a point where it can begin duplicating the trajectory of cancer research. "It is now clear that most autism, like cancer, has a genetic basis and that while rare forms of both are caused by mutations in single genes, many defective genes in a pathway generally need to work together to produce disease," he says.

By determining the mechanisms by which malfunctioning genes affect the common neural pathways linked to autism, Gargus believes, it will be possible to design or repurpose existing drugs to normalize the function of those channels. Like cancer therapy, this may well require a synergistic cocktail of drugs that act on distinct but interacting targets.

Three separate drug discovery efforts by UC Irvine neuroscientists involving these neural pathways are being integrated into CART.

Pharmacology professor Kelvin Gee is testing compounds that stabilize the firing rate of brain cells; psychiatry & human behavior professor Gary Lynch is using molecules called ampakines to increase growth factors in brain cells that allow for normal signaling; and Daniele Piomelli, the Louise Turner A
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Contact: Tom Vasich
tmvasich@uci.edu
949-824-6455
University of California - Irvine
Source:Eurekalert

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