Huntington's Disease Findings Pave Way For Potential Cure

New York, N.Y., August 8, 1997- Nearly 20 years ago, Columbia University researchers observed brain cell changes in Huntington's disease patients. Today, the significance of those changes has finally been explained--and the new findings may lead to a treatment or cure for the progressive, degenerative brain disease.

In the August 7 issue of Cell, Dr. Gillian Bates of the Division of Medical and Molecular Genetics, Guy's Hospital, London; Dr. Stephen W. Davies of the Department of Anatomy and Developmental Biology, University College London; Dr. Hans Lehrach and Dr. Erich Wanker of the Max Planck Institute for Molecular Genetics in Berlin, and colleagues have described the changes with unprecedented clarity. The cellular and biochemical mechanisms they have discovered could be a starting point for the development of new drugs that could interrupt the progress of Huntington's disease or prevent it in people at risk.

The gene for Huntington's disease (HD) was discovered in 1993 through the collaborative efforts of six research teams led by Nancy S. Wexler, PhD, Higgins Professor of Clinical Neuropsychology at the College of Physicians and Surgeons of Columbia University in New York and President of the Hereditary Disease Foundation in Santa Monica. Under the direction of Wexler, the Foundation has formed the Cure Huntington's Disease Initiative to accelerate progress from research to therapy.

Symptoms of Huntington's disease include abnormal movements, moodiness, depression, slurring and loss of speech, and, finally, mental incompetence, incontinence, confinement, and unremitting decline over 10 to 20 years. This progressive degenerative brain disease affects about 30,000 Americans; 150,000 more have a genetic risk for developing the illness. The cause is a mutation in a gene located on chromosome 4: Instead of having molecule groups that repeat a small number of times, they repeat 40 or more times and produce a defective form of th

Contact: Carolyn Conway
Columbia University Medical Center

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