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NYSCF and Columbia researchers demonstrate use of stem cells to analyze causes, treatment of diabetes
Date:6/17/2013

d the GCK mutation without introducing any potentially harmful additional DNA.

"Generation of patient-derived beta cells with gene correction could ultimately prove to be a useful cell-replacement therapy by restoring patients' ability to regulate their own glucose. This result is truly exciting," said Susan L. Solomon, Chief Executive Officer of The New York Stem Cell Foundation.

The researchers also used an electron microscope to assess beta cells for insulin content by counting granulespackages that store insulin for release. Even though all beta cell types had a similar number of granules, complete loss of function of the GCK gene was associated with decreased beta-cell production.

"These studies provide a critical proof-of-principle that genetic characteristics of patient-specific insulin-producing cells can be recapitulated through use of stem cell techniques and advanced molecular biological manipulations. This opens up strategies for the development of new approaches to the understanding, treatment, and, ultimately, prevention of more common types of diabetes," said co-senior author Rudolph Leibel, MD, Christopher Murphy Memorial Professor of Diabetes Research, Columbia University Medical Center, and Director, Division of Molecular Genetics, and Co-Director of the Naomi Berrie Diabetes Center.

The other authors are: Linshan Shang and Hector Martinez of the New York Stem Cell Foundation Research Institute; and Matthew Freeby, Mary Pat Gallagher, Thomas Ludwig, Liyong Deng, Ellen Greenberg, Charles LeDuc, Wendy K. Chung, and Robin Goland of the Division of Molecular Genetics, Department of Pediatrics, and Naomi Berrie Diabetes Center at Columbia University.


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Contact: David McKeon
dmckeon@nyscf.org
212-365-7440
New York Stem Cell Foundation
Source:Eurekalert

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