Columbia researchers receive $3 million to combat genetic killer of infants ...( l be joining the institution. Dr. Hend...),Health

l be joining the institution. Dr. Henderson will establish a Center for Motor Neuron Biology and Disease at CUMC focused on high-level translational motor neuron research. Working collaboratively with each laboratory and contributing to the translational research process, the new center will facilitate transfer of screening techniques, active chemical molecules and knowledge of disease causing pathways.

Darryl De Vivo, M.D., the Sidney Carter Professor of Neurology at CUMC and the director of the SMA Clinic, will direct the next step toward bringing new therapies to SMA patients through clinical trials. Dr. De Vivo is leading a multi-center network of investigators the Pediatric Neuromuscular Clinical Research Network that includes CUMC, Harvard University and the University of Pennsylvania researchers, as well as data management from the University of Rochester. Beginning in 2005, Dr. De Vivo and the network will conduct clinical trials in SMA patients with drugs that are currently under study as well as any compounds newly discovered by Dr. Stockwell, Dr. Monani and others. It is hoped that one more of these drugs candidates will significantly reduce the devastating effects of SMA in patients.

James L. Manley, Ph.D., a molecular biologist and Julian Clarence Levi Professor of Life Sciences in the Department of Biological Sciences at Columbia University is studying, together with his colleague Tsuyoshi Kashima, M.D., the basic biology of the SMN gene to find ways in which to recover its activity in patients. He is interested in how information in the chromosome is converted into messenger RNA to produce the SMN protein and is trying to understand the defect in that process that results in SMA disease.

Thomas Jessell, Ph.D., Investigator at the Howard Hughes Medical Institute and Director of the Neuroscience Institute at Columbia University, is focused on defining the molecular mechanisms that control motor neuron differentiation an
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Contact: Craig LeMoult
cel2113@columbia.edu
212-305-0820
Columbia University Medical Center
4-Mar-2005

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