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Disease in a dish – modeling human diseases using induced pluripotent stem cells

Tuesday, October 09, 2012 — Concurrent Symposia Session I

3:00 p.m. – 5:00 p.m.

Balcony A


  • Manfred Boehm, NHLBI
  • Kapil Bharti, NINDS


Our current understanding of human genetic disease pathogenesis is limited by the failure of animal models to completely recapitulate all the features of human genetic disease. Our ability to reprogram somatic cells into induced pluripotent stem cells (iPSCs) provides unique opportunities for studying the molecular and cellular mechanisms underlying the initiation and progression of human diseases. Patient cells can be reprogrammed into iPS cells, which, in turn, can be differentiated into the relevant cell type(s) specific to that disease. In addition to serving as models for the identification of novel disease mechanisms, these lineage-differentiated iPSC-derived cells can be used for high throughput screening of small molecule for the development new treatment strategies and for environmental toxin screening to investigate cell type-specific impact of toxins on cellular homeostasis. With this proposal, we would like to provide an overview of the new and exciting developments within the NIH campus utilizing iPSC-technology for investigating disease mechanisms, therapeutic compound screening, and preclinical treatment strategies.

Modeling of neuroinflammatory diseases using an autologous system
Avindra Nath, NINDS

Efficient production of iPSCs from patients with Chronic Granulomatous Disease and genetic correction of the oxidase defect in neutrophils differentiated from these iPSCs
Harry Malech, NIAID

Microglia: Friend and foe of neurologic diseases
John Park, NINDS

Differential susceptibility to ozone-induced lung inflammation maps to mouse chromosome 17: role of Notch receptors FARE Award Winner
Kirsten Verhein, NIEHS

Bone marrow failure, telomeres and iPSC – Syndrome, cause and model
Thomas Winkler, NHLBI

Drug screening using stem cell derived cellular disease models
Marc Ferrer, NHGRI

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