Stem cells, reprogramming and personalized medicine: promise, problems, reality
Rudolf Jaenisch, Professor of Biology, MIT Founding Member, Whitehead Institute for Biomedical Research
Description: After years of relentless lab work, rising and falling expectations, and the challenge of a sometimes hostile public, Rudolf Jaenisch says, "The scenario that looked like a fantasy has come closer to reality. We can study complex human diseases in a Petri dish and potentially contribute to therapy." In this lecture, Jaenisch describes the history of stem cell research and recent progress -- a story in which he has played a central role.
Jaenisch relates how stem cell science emerged from other significant work in molecular biology, such as his own breakthroughs with transgenic mice. Early in his career, Jaenisch began using viruses to study development, especially the puzzling way genes worked to switch on key sequences of growth. Jaenisch helped explicate some of the mechanisms of gene expression, or epigenetics, the system that makes genes readable or unreadable. In a "simple analogy for non"biologists," Jaenisch says that genetic information without epigenetics is like a Shakespeare speech without punctuation and spaces -- information content that's hard to read.
Jaenisch's lab learned how to manipulate genes essential for development, and this led to exploration of embryonic stem cells. Stem cells have the potential to develop into any type of cell (called pluripotency). Scientists imagined that genetic manipulation would permit shaping these cells into tissues that could be used for "customized tissue repair in degenerative diseases, and maybe for rejuvenation and prolongation of life." Enormous hype, and hope, immediately surrounded the concept of regenerative medicine. Jaenisch displays some tabloid advertisements falsely describing stem cell cures for dread diseases.
A subsequent advance, a technique called nuclear transfer that involves replacing the egg's original genetic material with that of a somatic donor cell, created even greater opportunities in the stem cell field. For Jaenisch, this held out even greater promise for therapeutic applications of stem cells. But for others, it raised the specter of human reproductive cloning, generating "enormous debate."
The field took another momentous turn when researchers learned how to induce pluripotent cells without the egg, says Jaenisch. Skin cells could become beating heart muscle, for instance. Recently, Jaenisch and his team have been at work reprogramming cells in culture, and attempting to resolve remaining challenges to customized stem cell therapy. These include eliminating the possibility of introducing viruses into growing cells, precisely integrating the correct genes in human induced pluripotent stem cells, and identifying the best kinds of donor cells for culturing. His current research involves modeling Parkinson's disease, exposing cells in culture to the kind of stress a human patient might experience, and "telescoping the aging process."
About the Speaker(s): Rudolf Jaenisch is one of the founders of transgenic science (gene transfer to create mouse models of human disease). His lab has produced mouse models leading to new understanding of cancers and various neurological diseases.
He received his doctorate in medicine from the University of Munich in 1967. He came to the Whitehead from the University of Hamburg in Germany, where he was head of the Department of Tumor Virology at the Heinrich Pette Institute.
Jaenisch received the 2002 Robert Koch Prize for Excellence in Scientific Achievement. In 2003, he was awarded the Charles Rodolphe Brupbacher Prize for basic research in oncology and was elected a member of the National Academy of Sciences.
Jaenisch is a fellow of the American Academy of Arts and Sciences and the American Academy of Microbiology, and a member of the American Association for the Advancement of Science.
Host(s): Office of the President, Office of the President
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