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The official announcement of Neurologix regarding the first 12
patients (Friday, 04 November 2005)
Gene therapy based on GAD gene transfer was well tolerated and
effective in improving parkinsonian symptoms in the first 12 patients
monitored for 4 to 12 months. The improvement regarded both motor
symptoms and the activities of daily living. These are the
preliminary results of a clinical trial sponsored by the American
Company Neurologix in 12 patients with advanced Parkinson's disease
who underwent surgery for the positioning of a catheter that enabled
gene transfer into the subthalamic nucleus of the brain. The gene
codes the most potent inhibitory neurotransmitter of the brain, GABA.
The hypothesis was that the increase in its availability would result
in the correction of the imbalance in nervous circuits due to the
death of dopaminergic neurons. The preliminary clinical data and the
neuroimages indicate that it does really correct the imbalance, at
least partially. The study is still ongoing and will end in 2006.
Should these results be confirmed, the researchers, who are the
founders of Neurologix, will search for funds to develop gene therapy
for the treatment of advanced Parkinson's disease.
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