October 10, 2002
First Human Parkinsons Gene Therapy Trial To Start

This is exciting:

The first human clinical trial of a gene therapy treatment for Parkinson's disease is set to begin in the US, following successful results in animals.

Share |      Randall Parker, 2002 October 10 11:59 AM  Biotech Therapies

Rachel Clarke said at October 13, 2002 1:21 AM:

Please send updated information

Kamran Usmani said at October 14, 2002 10:53 AM:

The tremors and other motor symptoms characteristic of Parkinson's disease are caused by destruction of nigral dopamine neurons of the subthalamic nucleus and excessive activity of output nuclei. In October 11 Science, Jia Luo and colleagues at the University of Auckland, New Zealand show that treatment with a glutamic acid decarboxylase (GAD) gene switches the subthalamic nucleus (STN) to inhibitory neurotransmission and reduces symptoms in a Parkinson's disease rat model (Science, 298:425-429, October 11, 2002).

Luo et al. injected adeno-associated viral vectors containing the gene that encodes GAD — which catalyzes synthesis of the inhibitory neurotransmitter GABA — into the STN of rats. They observed that following injection the excitatory glutamatergic neurons in the STN started producing mixed inhibitory responses associated with GABA release. This phenotypic shift resulted in strong neuroprotection of nigral dopamine neurons and rescue of the Parkinsonian behavioral phenotype.

"This strategy suggests that there is plasticity between excitatory and inhibitory neurotransmission in the mammalian brain that could be exploited for therapeutic bene?t," conclude the authors.

Kris said at October 15, 2002 7:22 PM:

I keep hearing about the Gene Therapy trials starting by the end of the year but where are they doing this study? Who is doing it?

Randall Parker said at October 16, 2002 9:32 AM:


Some details are in a later post I made. Short answer: Jefferson College in Philadelphia.

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