Peter W. Stacpoole, Ph.D., M.D.
Professor
Medicine and Biochemistry & Molecular Biology

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Causes and treatment of genetic mitochondrial disease
Inborn errors of mitochondrial metabolism may be due to mutations in either nuclear or mitochondrial DNA. The devastating consequences of these diseases are considered to be due primarily to disruption of both oxidative phosphorylation and the efficient conversion of substrate fuels into ATP. The cardinal clinical manifestations include lactic acidosis, progressive neurological and neuromuscular degeneration, exercise intolerance and cardiac disease, although virtually any organ system can be affected.

Our multidisciplinary team of mass spectrometrists, neuroscientists, pharmacologists, biochemists and clinicians has developed complementary approaches toward elucidating the pathogenesis of and developing new treatments for genetic mitochondrial diseases. These studies include the development of gene "knockdown" animal models of using siRNA technology; neurobehavioral, molecular and biochemical characterization of the resulting phenotype; and novel pharmacological and gene therapy paradigms to "rescue" the disease model as a prelude to conducting clinical trials in humans. We use cultured human animal cells to investigate downstream biochemical consequences of discrete mitochondrial gene mutations and the effects of putative therapeutic agents on the peripheral and central nervous systems. We translate this new knowledge into designing clinical protocols conducted in the GCRC in affected children, for whom there is currently no proven effective treatment. 		Status:
Not Accepting New Students This Year

Contact Information:
office: M-242
lab: M2-240 and M2-243
phone: (352) 392-2321
email: peter.stacpoole@medicine.ufl.edu
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Biography:
Professor Peter W. Stacpoole earned his PhD in pharmacology at the University of California, San Francisco in 1971. After a postdoctoral fellowship in the Metabolic Research Unit at UCSF, he attended Vanderbilt University, where he received his MD (1976) and subsequent training in internal medicine with a subspecialty in endocrinology and metabolism. He joined the faculty of the University of Florida in 1980 and rose to the rank of Professor in 1988. He was named Director of the General Clinical Research Center (GCRC) that year and both the Director of UF's Clinical and Translational Science Institute and Associate Dean for Clinical Research and Training in the College of Medicine in 2006. He was awarded the College of Medicine's Clinical Science Award in 2003 and UF Research Foundation Professorship from 2003-2006. Dr. Stacpoole was elected a Distinguished Fellow in the Royal College of Physicians in 2002.

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