Goldmine of Drug Targets: Study Finds Region in Brain Containing Genes Linked to Autism

Los Angeles, CA (Scicasts) — UCLA scientists have discovered that an overlooked region in brain cells houses a motherlode of mutated genes previously tied to autism. Recently published in Neuron, the finding could provide fresh drug targets and lead to new therapies for the disorder, which affects one in 68 children in the United States. “Our discovery will shed new light on how genetic mutations lead to autism,” said principal investigator Dr. Kelsey Martin, interim dean and a professor of biological chemistry at the David Geffen School of Medicine at UCLA. “Before we can develop an effective therapy to target a gene, we must first understand how the gene operates in the cell.” The UCLA team focused on a gene called Rbfox1, which regulates how the cell makes proteins —…


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