Thursday, March 17, 2011

Is Gene Therapy for Stuttering Near?

A fascinating article appearing in the Wall Street Journal ("Gene Therapy Raises Hopes for Parkinson's Treatment," March 17, p. A5) discusses a phase II trial for a Parkinson's gene therapy treatment. While in many ways, Parkinson's disease is the opposite of stuttering--too little dopamine vs. too much--the specific treatment may be applicable to stuttering.

Patients with Parkinson's also lose GABA. The therapy involves delivering a glutamic acid decarboxylase (GAD) gene to the bilateral subthalamic nuclei (STN) in the brain via an inert virus. The gene is responsible for making the chemical GABA and the therapy is said to improve motor function in Parkinson's victims.

Since some drugs such as benzodiazepines and pagoclone act to enhance the GABAergic system, it appears that the therapy cited above for Parkinson's might function also to increase GABA levels in people who stutter.

The controlled double blind phase II trials have been quite successful and Neurologix, Inc. is working with the USFDA to launch a phase III trial. And gene therapy for stuttering may be closer than you think.

A website providing more info can be found at:

A news release from Neurologix can be found at:

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