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Reprogramming patients' cells offers powerful new tool for studying, treating blood diseases
Date:7/30/2013

studies. The facility's goal is to develop and maintain standardized iPSCs lines specific to a variety of rare inherited diseasesnot only DBA and JMML, but also dyskeratosis congenita, congenital dyserythropoietic anemia, thrombocytopenia absent radii (TAR), Glanzmann's thrombasthenia and Hermansky- Pudlak syndrome.

A longer-term goal, added Weiss, is for the iPSC lines to provide the raw materials for eventual cell therapies that could be applied to specific genetic disorders. "The more we learn about the molecular details of how these diseases develop, the closer we get to designing precisely targeted tools to benefit patients."


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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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