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NIH grants to Children's Hospital will advance novel stem cell treatments for blood disorders
Date:2/16/2010

odel system for understanding how blood disorders develop. "Many blood diseases are difficult to study in patients, in terms of the exact mechanisms by which cells develop abnormally," said Weiss. "We will investigate iPSCs in models of two pediatric disorders, with the goal of using our improved knowledge of cell biology to devise treatments."

Weiss's group will focus on blood diseases associated with Down syndrome. Children with Down syndrome are at higher risk for transient myeloproliferative disorder, a precursor of leukemia, as well as for acute megakaryoblastic leukemia. Using animal models, the researchers will stimulate iPSCs to mimic disease processes seen in Down syndrome, in hopes of discovering ways to prevent these forms of leukemia.

A third goal of Weiss's project is to collect tissues from patients, then reprogram their cells into iPSCs and develop them into tissue banks for specific diseases. In this effort, his team will concentrate on a genetic blood disorder called Diamond Blackfan anemia.

Weiss's team at Children's Hospital will partner with scientists at two other institutions in the region: Pennsylvania State University, in State College, Pa., and the Coriell Institute for Medical Research, in Camden, N.J.

"The successful completion of these grants was in large part based on the recent establishment of the Human Embryonic Stem Cell Core at Children's Hospital," said Poncz. "These two grants illustrate the promising future that stem cell biology holds, not only for research purposes and for hematologic and oncologic disorders, but for a wide range of diseases that presently have suboptimal therapies. The future of stem cell therapy may be limited only by our imagination."


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Contact: John Ascenzi
Ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
Source:Eurekalert

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