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September 2006
Children's Research Institute News Brief
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Bernard L. Maria, MD, MBA
Executive Director
Darby Children's
Research Inst. |
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Inderjit Singh, PhD
Scientific Director
Darby Children's
Research Inst. |
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DCRI laboratory is originator of many collaborative research efforts
Pioneering research in the basic science laboratories of Dr Inderjit Singh continues to lead to further investigations,
grants and discoveries throughout MUSC and the medical world.
"We have been able to translate much of the basic science work performed in Dr. Singh's lab into clinical medicine with great results," says Dr. Lyndon Key.
Dr. Singh's lab conducts studies that have an impact on cerebral palsy, Down syndrome, spinal cord injury, MS, and stroke.
Many other investigations and human trials have resulted from this research, says Dr. Singh, a professor of pediatrics and
director of the division of Development Neurogenetics.
The DCRI lab is usually involved with 10 different studies at any one time.
"The DCRI is investigating the mysteries of brain functions," says Dr. Singh. "We're studying how different cell types in the
brain communicate to derive a desired function, and how the loss of these functions results in a disease."
The lab conducts studies of the brain under normal as well as disease conditions as a result of loss of function and brain cells.
Ideally, lost cells should be replaced with new cells.
Embryonic stem cells are considered the best replacement for damaged cells and tissue, yet their use is controversial, notes Dr. Singh.
Scientists recently discovered that the brain is able to regenerate cells to compensate for such loss.
"Stem cells for different cell types of the brain have been found to be present even in the adult human brain," he explains.
"Unfortunately, these stem cells are vulnerable and unstable under disease conditions, restricting their ability to regenerate and repair damage."
Dr. Singh's lab is investigating ways to protect the stem cells in the diseased adult brain with drug therapies. "Once stem cells
are protected, they can become mature functioning brain cells and thus compensate for the loss," he says.
Scientists in Dr. Singh's DCRI group are evaluating drug therapies in a number of disease conditions, including leukodystrophies,
cerebral palsy, perinatal hypoxia, brain /spinal cord injury, Down syndrome, MS and stroke.
These complex studies bring together investigators from many different disciplines, including biochemists, cell biologists, molecule
biologists and physicians with pediatrics, neurology, pathology and OB/GYN backgrounds.
"These collaborative studies have made major advances in enabling us to understand the patho-biology of these diseases," credits Dr. Singh.
His lab uses tissue cultures as well as animal models to create drug therapeutics, some of which are now being tested or have received
funding to be tested in humans.
For instance, based on investigations initiated by Dr. Singh's lab, the FDA has funded a human study (a collaboration between MUSC and the
University of Pennsylvania) to test the efficacy of statins, cholesterol-lowering drugs, in children with Type I diabetes.
"This Type I diabetes study resulted from studies we conducted on MS," notes Dr. Singh. A collaborative study between investigators from MUSC,
Yale University, and University of Colorado had reported efficacy of simvastatin treatment in MS. Now this drug treatment is being tested in a
study with a larger group of MS patients, headed by an investigator from University of California at San Francisco.
The NIH also recently funded a nearly $2 million study to test the efficacy of NAC for maternal infection, based on initial investigations
performed in Dr. Singh's lab. (See feature story).
More studies are expected to result from the lab's basic science discoveries, including investigations of sickle cell disease, neonatal maternal
morbidity, mortality caused by preeclampsia, renal disease and autoimmune disease.
"Discoveries made here in our lab with children's diseases translate to children, the Children's Hospital, and beyond," says Dr. Singh. "They are
ultimately beneficial in treating and/or preventing adult diseases such as MS and stroke."
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