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

In addition to boosting the supply of platelets to carry out their usual biological roles, the researchers also seek to customize them as drug delivery vehicles. In mouse studies, for instance, Poncz's team previously treated the bleeding disorder hemophilia by loading platelets with the clotting factor that is deficient in that disease. "In addition to investigating platelets for treating hemophilia in people, we will investigate their potential role in delivering other bioactive proteins to sites of vascular injury," said Poncz. "For instance, platelets might deliver an enzyme called urokinase to selectively disintegrate blood clots."

Second grant will investigate induced pluripotent stem cells A separate grant from the NHLBI also supports stem cell research, but focuses on a more recently discovered type of cell. This two-year, $997,000 grant was awarded to hematologist Mitchell Weiss, M.D., Ph.D. The Grand Opportunity (GO) Grant, funded by the American Recovery and Reinvestment Act, is part of an NHLBI program to support novel research designed to quickly advance an area of biomedicine in significant ways.

Scientists demonstrated in 2007 that they could reprogram human somatic cells (the vast majority of cells that are not sperm or egg cells) into a pluripotent statethe capacity to develop into other types of human cells. In this project, Weiss and colleagues will manipulate induced pluripotent stem cells (iPSCs) into becoming hematopoietic, or blood-forming, cells.

"These cells represent a potentially remarkable tool for custom-fitting new tissue to an individual patient," said Weiss. "Because they originate from an individual patient's cells, they will not be rejected as foreign by the patient's immune system." But much work remains to be done in fully characterizing how iPSCs develop, and in understanding how they may differ from both hESCs and from typical blood cells.

Weiss will also investigate iPSCs as a powerful new m
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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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