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Foundation grants bolster autism, dystonia research

Sep. 4, 2014, 10:19 AM

Vanderbilt University researchers have received multi-year grants from two private foundations to investigate key signaling proteins that could lead to novel treatments for autism spectrum disorder (ASD) and dystonia, a movement disorder.

“Both awards exemplify the multidisciplinary, collaborative research needed to advance biomedical objectives,” said the grants’ principal investigator, Randy Blakely, Ph.D., Allan D. Bass Professor of Pharmacology. “They also underscore the importance of private foundation support for research with the goal of accelerating research toward novel therapeutics.”

Randy Blakely (Vanderbilt University)
Randy Blakely, Ph.D.

The focus of the three-year grant from the Simons Foundation Autism Research Initiative is the p38alpha MAPK signaling pathway, which Blakely proposes links the body’s immune response to the neurotransmitter serotonin.

MAPKs, for mitogen-activated protein kinases, are enzymes that regulate the body’s response to stress stimuli, among other roles.

Prenatal infection, chronic inflammation, and abnormal immune responses have been associated with an increased risk for ASD, as have altered brain and blood serotonin levels. The Vanderbilt team is currently exploring how p38alpha MAPK and serotonin signaling intersect using genetic mouse models of ASD developed by the Blakely lab.

Collaborators on the grant include Blakely lab postdoctoral fellow Matthew Robson, Ph.D., and Mark Boothby, M.D., Ph.D., professor of Pathology, Microbiology and Immunology, and Medicine.

The two-year award by the Dystonia Medical Research Foundation will support efforts to capitalize on Blakely’s research into the role of presynaptic choline transporters in sustaining signaling by the neurotransmitter acetylcholine.

Dystonia is a movement disorder that affects hundreds of thousands of children and adults in the United States. Treatments for dystonia include medications that reduce the actions of acetylcholine in the control of motor activity.

Blakely, along with Pharmacology graduate student Elizabeth Ennis, and Craig Lindsley, Ph.D., William K. Warren Jr. Professor of Medicine, are developing novel compounds that limit neurotransmitter activity by reducing uptake of choline, the essential precursor to acetylcholine, by transporters in nerve terminals.

The research reflects key infrastructure investments supported by the Transgenic Mouse/ESC Shared Resource, the Laboratory for Neurobehavior, the Vanderbilt Conte Center, which Blakely directs, and the Vanderbilt Center for Neuroscience Drug Discovery to generate and study the behavior of genetically modified mice, he said.

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