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Cedars-Sinai research team awarded $1.9 million from CIRM to develop new tools and technologies
Date:2/2/2011

will stimulate SMN protein development in motor neurons. To that end, they will convert patient skin cells to powerful induced pluripotent stem cells and then motor neurons with SMN1 mutation, but still containing the intact SMN2 gene.

Then, the team will begin testing compounds to see if any help increase motor neurons' production of the SMN protein through the SMN2 gene.

"Pharmaceutical and biotech companies have comprehensive libraries of chemically diverse compounds, some of which potentially could be developed into drug treatments to help SMA patients," Sareen said. "We will be able to test these drug libraries relatively quickly using our high-content screening approach. We hope to identify novel compounds that target the SMN2 gene specifically in patient motor neurons. Any drug lead "hits" we get can be quickly repurposed and pursued, bringing us so much closer to finding a treatment for this debilitating childhood disease."

Another team led by Dan Gazit, Ph.D., DMD and Zulma Gazit, Ph.D. is working with Gamma Medica-Ideas, Inc., which was granted $1.5 million to develop a new imaging technology that for the first time would enable doctors to monitor stem cell treatments in patients in real time.

Their project is to combine magnetic resonance imaging (MRI), which uses magnetic field and radio waves to create images of organs and tissues, with single-photon emission computerized tomography (SPECT), which uses a radioactive substance and a special camera to produce three-dimensional images. The scientists believe the new imaging system will be most applicable at first for muscle, tendon, ligament, cartilage and intervertebral disc regeneration. In the future, however, the new imaging system is likely to be developed to monitor stem cell treatments for other tissues, particularly in the heart and brain.

"A common challenge facing the use of stem cells to regenerate damaged tissue is that we have no im
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Contact: Sally Stewart
Sally.stewart@cshs.org
310-248-6566
Cedars-Sinai Medical Center
Source:Eurekalert

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