iPS Cell Technology, Gene Editing and Disease Research

CCR Eminent Lecture Rudolf Jaenisch, a Whitehead Institute Founding Member, focuses on understanding epigenetic regulation of gene expression (the biological mechanisms that affect how genetic information is converted into cell structures but that don’t alter the genes in the process). Most recently, this work has led to major advances in our understanding of embryonic stem cells and “induced pluripotent stem” (IPS) cells, which appear identical to embryonic stem cells but can be created from adult cells without using an egg. In 2007, the Jaenisch lab was one of three labs worldwide that reported successfully taking cells from mouse tails and reprogramming them into IPS cells, by over-expressing four master gene regulators. Later that year, the lab followed up by further manipulating IPS cells to treat sickle-cell anemia in mice, the first proof in principle of therapeutic use of such cells. In 2008, the lab reported that neurons derived from IPS cells successfully integrated into fetal mouse brains and reduced symptoms in a Parkinson’s disease rat model. In another experiment, researchers demonstrated that fully mature, differentiated mouse B cells can be reprogrammed to IPS cells. Researchers are now studying ways to optimize the creation of IPS cells, including finding alternatives to the potentially cancer-causing retroviruses used to transform the adult cells into IPS cells. In the long run, IPS cells offer major promise for use in regenerative medicine, potent...
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