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2001 Genetics Prize

Rudolf Jaenisch

Laureate Profile

Rudolf Jaenisch is a Founding Member of Whitehead Institute and a pioneer of transgenic science, in which researchers alter an animal's genetic makeup to produce a variant of a human disease. Jaenisch has focused on creating transgenic mice that enable his lab to study forms of cancer and neurological diseases that have long baffled researchers.

Jaenisch's first breakthrough occurred in the 1970s when he demonstrated for the first time that foreign DNA could be integrated into the DNA of early mouse embryos; mice derived from these embryos carried the foreign genes in all of their tissues. Subsequently, Jaenisch injected retrovirus into early mouse embryos and showed that leukemia DNA sequences had integrated not only into the mouse genome but also to its offspring. These mice were the first transgenic animals in history.

Jaenisch also is a leader in the field of therapeutic cloning, also called nuclear transfer, in which the genetic information from one cell is transplanted into an unfertilized egg from which DNA has been removed. When placed in a Petri dish, this egg develops into a blastocyst from which stem cells can be taken. Jaenisch's therapeutic cloning research occurs exclusively with mice, but he advocates using the same techniques with human cells so that we may one day develop therapies for diseases that are currently untreatable. Conversely, Jaenisch opposes human reproductive cloning, in which the egg is placed not in a Petri dish but into the uterus of a female, in the hope that it will develop into a fetus. Recently, using mice as a model, Jaenisch demonstrated that it is possible to procure embryonic stem cells without harming a viable embryo. Using a technique called "altered nuclear transfer," Jaenisch created an embryo-like entity that was genetically incapable of implanting in a uterus, and that also had certain structural deficiencies. Although this entity was not a viable embryo, it did yield perfectly healthy embryonic stem cells. If this research is successfully repeated in human cells, it might provide a solution to the ethical debate.

Today, the over-arching goal of Jaenisch's lab is to understand what scientists call epigenetic regulation of gene expression. This refers to the biological mechanisms that affect how genetic information is converted into cell structures but that don't alter the genes in the process. Jaenisch's lab is also investigating epigenetic mechanisms for certain types of cancer and for brain development, where much of his work focuses on how conditions such as Rett Syndrome occur.
