(PHILADELPHIA) - The University of Pennsylvania has received a $2 million grant from the W.M. Keck Foundation of Los Angeles for a pioneering study on the genomics of Parkinson's disease. The Keck Foundation's program supports basic biomedical research and the development of pioneering new technologies.
The grant will permit the development of technology to study the basic cell biology of Parkinson's disease. This interdisciplinary project involves a diverse team of researchers from Penn's Genomics Institute: molecular biologist James Eberwine, PhD, Department of Pharmacology; computational scientist Junhyong Kim, PhD, from the Department of Biology; imaging experts Philip Haydon, PhD and Jai-Yoon Sul, PhD, from the Department of Neuroscience; robotics expert Vijay Kumar, PhD, from the Department of Mechanical Engineering; and biological modeling expert David Meaney, PhD, from the Department of Bioengineering.
"The scientific scope of such an interdisciplinary effort has traditionally been difficult to fund through standard NIH mechanisms," notes Eberwine, the project's principal investigator. "The foresight of the Keck Foundation in facilitating this and such efforts at other universities will undoubtedly contribute to the development of scientific innovation and therefore the improvement of the human condition."
Eberwine and colleagues will develop new approaches that target the multi-genic nature of Parkinson's disease. Parkinson's is a debilitating, progressive neurodegenerative disorder of the central nervous system in which dopamine-producing nerve cells die in those areas of the brain that coordinate voluntary movement and aspects of cognition. Dopamine is a neurotransmitter that permits selected neurons to communicate with one another. Decreased levels of dopamine cause a broad class of movement disorders, including muscular tremors and weakness.
The Keck grant will fund the cataloguing of the changes in gene expression that underlie the development of Parkinson's in individual live neurons and will utilize a newly developed technology for assessing genomic changes in live cells. The goal is to use this information to create neurons that reliably produce dopamine, under natural gene regulation within the cell. Eventually, such cells may prove to be useful in cellular therapeutics.
Current treatments for Parkinson's using transplanted cells are limited by the traditional one-gene-at-a-time approach to manipulating dopamine production. The new integrated approach developed by Eberwine and colleagues will identify the complex genetic nature of the disease and will be used in efforts to correct the multifaceted gene expression anomalies that underlie Parkinson's pathology.
More generally, most human diseases and syndromes manifest themselves through the dysregulation of multiple genes. The approaches being developed as part of this Keck grant will permit multiple genes to be manipulated in a predictable, naturally controlled manner. "These studies will provide the first truly functional genomics approach to understanding human disease and eventually, it is hoped, may provide novel therapeutic intervention strategies," says Eberwine.
The W.M. Keck Foundation, established in 1954 by William Myron Keck, founder of The Superior Oil Company, is one of the nation's largest philanthropic organizations with assets of more than $1 billion. In recent years, the Foundation has focused on five broad areas: Science and Engineering Research; Undergraduate Science and Engineering; Medical Research; Liberal Arts; and Southern California.
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Penn's School of Medicine is ranked #2 in the nation for receipt of NIH research funds; and ranked #3 in the nation in U.S. News & World Report's most recent ranking of top research-oriented medical schools. Supporting 1,400 fulltime faculty and 700 students, the School of Medicine is recognized worldwide for its superior education and training of the next generation of physician-scientists and leaders of academic medicine. The University of Pennsylvania Health System includes three hospitals, all of which have received numerous national patient-care honors [Hospital of the University of Pennsylvania; Pennsylvania Hospital, the nation's first hospital; and Penn Presbyterian Medical Center]; a faculty practice plan; a primary-care provider network; two multispecialty satellite facilities; and home care and hospice.