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The source of this article is Betterhumans: http://tinyurl.com/5rt4c

Parkinson's Gene Therapies Progress
One approach aims to halt disease's development, another aims to avoid
treatment side-effects
Betterhumans Staff
2/9/2005 5:32 PM


Two gene therapies for Parkinson's disease are showing promise at preventing
the disease's progression and avoiding side-effects of existing treatments.

The therapies are being studied by an international team from Lund
University http://www.lu.se/ in Sweden, the University of Cambridge
http://www.cam.ac.uk/in the UK and the University of Florida
http://www.ufl.edu/

One of the treatment approaches is aimed at halting Parkinson's disease at
its onset while the other is aimed at avoiding side-effects that occur when
treating the disease in its later stages.

Preventing damage

In a study of the first approach, researchers inserted corrective genes into
the brain of small monkeys called marmosets.

The genes helped to prevent brain damage by producing therapeutic levels of
a protein called glial cell line-derived neurotrophic factor (GDNF).

GDNF helps nourish brain cells and has attracted attention for treating
Parkinson's disease. Its use has been debated, however, since human trials
ended last year after trial-conductor Amgen cited safety concerns. Many
people with Parkinson's still want the treatment.

The gene therapy approach appears to produce more manageable levels of the
protein in the brain.

In tests with 31 monkeys, GDNF genes inserted into a region in the front
part of the brain called the striatum protected monkeys when drugs were used
to induce Parkinson's-like conditions by destroying dopamine-producing cells.

Seventeen weeks after receiving the drug, GDNF-treated monkeys showed
improvement in performing tasks and analysis of brain tissue showed the
animals' dopamine systems were spared.

Reversing abnormal movement

In the second approach, researchers used gene therapy to reverse abnormal
movements called dyskinesias in some rats.

The approach could combat flailing movements produced by Levodopa, a widely
used drug treatment for Parkinson's disease.

While Levodopa usually works well for several years, it can eventually start
causing movement problems.

It is hoped that gene therapy could prevent harmful fluctuations of L-dopa,
a precursor of dopamine.

In 33 rats with severe dopamine depletion who had limited use of their left
paw, gene therapy to provide a source of L-dopa caused complete recovery of
movement.

The studies are reported in the journal Brain and The Journal of Neuroscience.

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