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Patient-derived induced stem cells retain disease traits
Date:12/21/2008

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Ebert points out that the patient-derived iPS cells can offer scientific advantages over other approaches, including embryonic stem cells, for studying disease. In effect, the researchers can watch the unfolding of an accident that has already occurred, and the known clinical outcome the course and severity of the patient's disease should help them understand how the changes they see in the cells fit into the bigger picture of the disease.

"The development of human-derived SMA motor neurons is an important step forward for the SMA field, especially as a variety of therapeutic avenues are being examined," agrees SMA expert Christian Lorson, a professor of veterinary pathobiology at MU and an author on the paper. "To be able to investigate therapeutic activity in these cells, whether it be novel drugs, viral vectors, oligonucleotides, or a better understanding of disease pathology, the iPS SMA motor neurons represent an excellent disease-related context."

While complex and late-hitting disorders like Alzheimer's and Parkinson's diseases will be harder to model with iPS cells, the researchers say the approach should pave the way for studies of other genetic disorders, such as Huntington's disease. "We have to find better ways to model complex human diseases that are difficult to reproduce in animals," Svendsen says. "iPS cells represent a promising new research tool to reach this goal."

He credits the UW-Madison Stem Cell and Regenerative Medicine Center with facilitating the work, especially by drawing on the expertise of Yu and Thomson, who pioneered the technique, to create the iPS cells used in this study. "This is an example of how the center is working to collaborate on campus and off campus to bring these kind of things to fruition," he says.


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Contact: Clive Svendsen
cnsvendsen@wisc.edu
608-265-8668
University of Wisconsin-Madison
Source:Eurekalert

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