October 24, 2002
Huntington's Treatment Works In Mice

It is not clear from this report what technique they used to shut down the Huntington's gene. If they genetically engineered the mice to make the gene able to be shut down by pharmaceutical means then this result is only useful for studying the disease. But if they developed a drug that could shut down the regular regulatory region for this gene then the technique might be closer to a treatment usable on humans. If anyone knows exactly what they did do tell. Still, the fact that the toxic protein will clear out if the cell can be made to stop making it is good news for Huntington's patients:

Researchers have devised a clever genetic technique in mice that can regulate the production of the abnormal protein that causes Huntington's disease.

Ai Yamamoto, a researcher at Memorial Sloan-Kettering Cancer Center, created an animal model that exhibits all the signs of the lethal disease: brain damage and impaired movement. When the mutant gene is shut off, toxic protein deposits clear out and the animal improves substantially. Yamamoto presented her findings last week at the American Neurological Association's annual meeting in Manhattan.

Share |      Randall Parker, 2002 October 24 01:36 PM  Biotech Therapies


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