Goal 3: Advance Translational Research

Use isogenic iPS cells to advance Precision Medicine

The goals of Precision Medicine can be achieved if we determine the biological basis of disease-associated variants for NHLBI diseases. Advances in genetic research have yielded hundreds of disease-associated DNA polymorphisms, yet we lack robust methods to experimentally test their functional relevance in human cells. Determining the molecular and cellular basis of human phenotypic variation is one of the great challenges of medical research. An essential step towards this goal would be to establish human induced pluripotent stem (iPS) cells that have identical genetic (i.e., isogenic) backgrounds but differ only at putative disease–causing loci.

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Idea No. 489