Newswise — UAB researchers have won a highly prestigious grant from The Michael J. Fox Foundation (MJFF) to examine the function of a new gene connected to Parkinson's disease (PD). The $200,000 grant will allow investigators to delve into the role of kinase activity in the LRRK2 gene, which may play a major role in the progression of PD.
"The Michael J. Fox Foundation recently identified LRRK2 as a gene of great interest in PD. It is known to cause some cases of PD and may be involved in many cases of the disease," said David Standaert, M.D., Ph.D, professor of neurology and director of the UAB Center for Neurodegeneration and Experimental Therapeutics. "They challenged the scientific community to propose protocols to study the gene's role in Parkinson's and report findings within one year."
The UAB protocol is one of only four worldwide to be accepted for the Foundation's LRRK2 Critical Challenges in Parkinson's Disease Research Program.
UAB researchers say the university is uniquely positioned to meet this challenge and undertake this research.
UAB's Department of Neurology is already known internationally in Parkinson's research, under the leadership of department chair Ray Watts, M.D., and Standaert. The department recently recruited Andrew West, Ph.D., assistant professor of neurology, who is one of the world's leading experts on the LRRK2 gene. And, the UAB Gene Therapy Center, directed by David T. Curiel, M.D., Ph.D., professor of medicine, is an international leader in the development of virus-based delivery systems for gene therapy.
"UAB has the people, the expertise and the facilities to move quickly on this initiative," said Curiel. "The LRRK2 gene offers tremendous potential as a target for Parkinson's research. Our job now is to more fully understand the gene's role in PD and unlock that potential."
Research suggests that the protein product of the LRRK2 gene appears to have kinase activity, which regulates the function of other proteins and is typically involved in cell signaling pathways. Intriguingly, certain PD-associated gene mutations cause an increase in LRRK2 kinase activity, suggesting that this activity may play a central role in Parkinson's pathogenesis.
Among the first steps at UAB will be the genetic engineering of normal and mutant forms of the LRRK2 gene into viral vectors developed at the UAB Gene Therapy Center, a project that will be directed by Joel N. Glasgow, Ph.D., assistant professor of medicine. The vectors are used to introduce the gene into a mouse, producing a model of the disease. The model will help investigators determine if the kinase activity does play a significant role in the progression of PD.
"We hope to know within the year if, in fact, kinase activity is implicated in cell death," said Standaert. "If so, this opens a new doorway into the development of drugs that can regulate that activity."
Founded in 2000, The Michael J. Fox Foundation for Parkinson's Research is dedicated to ensuring the development of a cure for Parkinson's disease within this decade through an aggressively funded research agenda.. The Foundation has funded over $108 million in research to date, either directly or through partnerships.
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