Mouse Research Yields Gene Therapy Hope for Huntington's

The BBC recently reported on a successful effort in mice to treat Huntington’s with gene therapy. Huntington’s is a genetic disease that is incurable and results in the degeneration of the brain and eventual death among sufferers.

Researchers at the University of Iowa used gene therapy to shut off the affected gene entirely in mice. Shutting off the gene entirely in human beings is not an option, but there is another possibility. Human beings have two copies of this gene, and in most Huntington’s sufferers only one of the genes is defective. If researchers could turn off the defective gene, they could at least slow the progress of the disease.

So far, though, such gene therapy has shown limited success in slowing a similar disease human cells in laboratory conditions, but is still a long way from testing a gene therapy cure in human beings.


Gene therapy hope for Huntington’s. The BBC, March 13, 2003.

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