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NYP/Weill Cornell gene therapy clinical trial yields promising results for Batten disease
Date:5/13/2008

ntually die throughout the entire brain."

When this happens, children with the disease begin exhibiting neurological symptoms, starting around age 4, including impaired muscle coordination (ataxia), involuntary twitching (myoclonus), and speech and developmental disorders. A gradual decline in visual ability follows. Affected children generally become wheelchair-bound by the ages of 4 to 6 years and eventually become bedridden.

Because the disease is fatal early in life, there are only about 200 cases of the disease in the world at a given time. Subjects from around the world were carefully selected to take part in the trial.

Neurological surgeons from NewYork-Presbyterian Hospital/Weill Cornell Medical Center, led by Drs. Mark Souweidane and Michael Kaplitt, performed the gene therapy procedure. Six tiny holes were made in the skull of each subject, and then a liquid containing the healthy CLN2 gene, within the harmless adeno-associated virus (AAV), was injected into the brain.

"Before now, we had no hope of a therapy for Batten disease, but today we can say that there is some hope," says Dr. Crystal. "These results are not just promising for sufferers of the disease, but suggest that gene therapy can work and should be studied for other neurological disorders. Each gene in our body has the potential to become a target to study for human disease."


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Contact: Andrew Klein
ank2017@med.cornell.edu
212-821-0560
New York- Presbyterian Hospital/Weill Cornell Medical Center/Weill Cornell Medical College
Source:Eurekalert

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