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IPSC Cells for Screening and Therapy

Wednesday, October 26, 2011 — Concurrent Symposia Session IV

Noon – 2:00 p.m.

Balcony B


  • John O'Shea, NIAMS
  • Mahendra Rao, OD


The study of pluripotent stem cells is revolutionizing basic assumptions in biology. The rapidity with which the advances in stem cell biology will enter routine clinical practice remains to be determined, but there are few areas in biomedical research that are more exciting. The study of pluripotent stem cells affects virtually all scientists ranging from developmental and cell biologists to those interested in genomics and transcriptional and epigenetic regulation of gene expression. Most recently the topic of immunologic rejection of induced pluripotent stem cells has become an issue and will be of intense interest across the campus.

Using iPS Cell Derived Retinal Pigment Epithelium to Understand Eye Disease Mechanisms
Kapil Bharti, NINDS

An iPS Approach to the Analysis and Treatment of Smith-Lemli-Opitz Syndrome, a Disorder Caused by Defective Cholesterol Synthesis
Heiner Westphal, NICHD

Zinc Finger Nuclease-Mediated Safe-Harbor Targeted Gene Transfer in Patient iPSCs Functionally Corrects X-Linked Chronic Granulomatous Disease *FARE Award Winner
Colin Sweeney, NIAID

Development of iPSC for the Treatment of Neurologic Disorders
Mahendra Rao, OD

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