Research in Progress

Gene Therapy

Gene Therapy Successful in HD Rats

In early June 2003, researchers announced that certain proteins in the nervous system called neurotrophic factors may combat the degeneration of neurons associated with Huntington’s disease. Scientists used a harmless virus to transfer a neurotrophic factor called GDNF to the neurons of rats with HD in order to prevent the degeneration of these cells. This harmless virus can be used to deliver the neurotrophic factor into the nuclei of the neurons. To test the effectiveness of GDNF, each rat’s neurons were injected with one of two neurotrophic factors, GDNF or GFP, followed by injections below the skin of a harmful toxin. After the injection of the toxin, the rats were tested on a raised platform motor task, in which they had to find the platform and climb onto it. The motor skills of the GFP rats deteriorated, while the motor skills of the GDNF rats remained relatively normal. These results indicate that GDNF defends neurons against the degenerative effects of HD. However, researchers anticipate that further testing on HD mouse models will be necessary to determine if this type of gene therapy would be effective in humans.

Last Modified: 5-10-04

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