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NIH Research Festival 2012 Program - Research Festival - National ...

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Concurrent Symposia Session I<br />

Natcher Conference Center<br />

Balcony A<br />

Stem Cell Interest Group<br />

Disease in a dish—modeling human diseases<br />

using induced pluripotent stem cells<br />

Co-Chairs: Manfred Boehm, NHLBI; and Kapil Bharti, NINDS<br />

Our current understanding of human genetic disease pathogenesis is limited by the failure<br />

of animal models to completely recapitulate all the features of human genetic disease.<br />

Our ability to reprogram somatic cells into induced pluripotent stem cells (iPSCs) provides<br />

unique opportunities for studying the molecular and cellular mechanisms underlying<br />

the initiation and progression of human diseases. Patient cells can be reprogrammed<br />

into iPS cells, which, in turn, can be differentiated into the relevant cell type(s) specific<br />

to that disease. In addition to serving as models for the identification of novel disease<br />

mechanisms, these lineage-differentiated iPSC-derived cells can be used for high<br />

throughput screening of small molecule for the development new treatment strategies<br />

and for environmental toxin screening to investigate cell type-specific impact of toxins<br />

on cellular homeostasis. With this proposal, we would like to provide an overview of the<br />

new and exciting developments within the <strong>NIH</strong> campus utilizing iPSC-technology for<br />

investigating disease mechanisms, therapeutic compound screening, and preclinical<br />

treatment strategies.<br />

Avindra Nath, NINDS<br />

Modeling of neuroinflammatory diseases using an autologous system<br />

Harry Malech, NIAID<br />

Efficient production of iPSCs from patients with Chronic Granulomatous Disease and<br />

genetic correction of the oxidase defect in neutrophils differentiated from these iPSCs<br />

John Park, NINDS<br />

Microglia: Friend and foe of neurologic diseases<br />

Kirsten Verhein, NIEHS<br />

Differential susceptibility to ozone-induced lung inflammation maps to mouse<br />

chromosome 17: role of Notch receptors<br />

FARE Award Winner<br />

Thomas Winkler, NHLBI<br />

Bone marrow failure, telomeres and iPSC – Syndrome, cause and model<br />

Marc Ferrer, NHGRI<br />

Drug screening using stem cell derived cellular disease models<br />

Tuesday, October 9, <strong>2012</strong><br />

3:00 p.m.–5:00 p.m.<br />

19

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