In the first example of therapeutic cloning for treating a brain disorder, Lorenz Studer and colleagues have used dopamine neurons derived from cloned mouse embryonic stem (ES) cells to treat mice with a Parkinson-like condition. The study, published in the October issue of Nature Biotechnology, is only the second report of therapeutic cloning in animals.
A loss of dopamine neurons is a hallmark of Parkinson disease. Dopamine neurons derived from ES cells were previously shown to be effective in treating parkinsonian mice. But the cells in this earlier study expressed a transgene -- raising safety concerns -- and they had not been generated by cloning.
The goal of therapeutic cloning is to produce specialized cells needed to repair a failing organ from human ES cells generated by cloning the patient’s own cells. Unlike generic ES cells, such cloned ES cells and their specialized progeny would be genetically identical to the patient and could be transplanted without being rejected by the patient’s immune system. Although therapeutic cloning has great promise for treating many diseases, it has not yet been demonstrated in human patients.
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