Rapid drug discovery in genetic models of autism

  • Awarded: 2014
  • Award Type: Explorer
  • Award #: 313384

Over the past few years, studies have linked many genes to autism. With these discoveries comes the possibility of developing therapeutic approaches, but where to start? Alex Parker and his colleagues at the Centre Hospitalier de l’Universite de Montreal hope to use the worm C. elegans to find molecules that may be early drug development leads to help treat autism spectrum disorders.

C. elegans is a small, free-living worm that has been used to study many aspects of biology, and neuroscience in particular. Many genetic mutations linked to autism are associated with altered synaptic function — the passing of electrical and chemical signals between neurons — and neuronal circuit functioning. The regulation of synaptic and neuronal function has been extensively studied in C. elegans, and the worm’s genome encodes many genes that in humans are linked to autism.

Parker and his team developed rapid drug-screening methods that they plan to use to target synaptic dysfunction caused by autism mutations in worm models. They hope to find molecules that restore normal neuronal behaviors in simple genetic models of autism. These drugs may have clinical relevance and could aid further drug discovery and development for individuals with autism.

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