15 October 2009

Three-gene therapy for Parkinson's disease

Gene therapy has helped to relieve a number of symptoms of Parkinson's disease in macaquesDmitry Tselikov, Compulenta
With the help of gene therapy, French scientists have achieved a significant improvement in the motor functions of macaques with symptoms of Parkinson's disease.

There were no side effects caused by modern methods of treatment.

The simultaneous inclusion of three genes allowed certain brain cells to take over the production of the dopamine mediator. It is the low level of this substance that causes motor problems, which are the hallmark of Parkinson's disease.

The three above–mentioned genes have already been used separately in gene therapy, but never together. As a result of the experiment, the dopamine level reached half of the norm. The effect persisted for 44 months. At the same time, dopamine began to be produced by cells that usually do not do this, which caused concern among scientists. And although no complications were noticed in monkeys, the effect of the proposed method on humans needs to be clarified further.

In addition to tremors and other motor problems, patients often develop dementia, behavioral problems, depression, anxiety, heart problems, loss of sense of smell, constipation and sexual dysfunction. Most of the symptoms are again associated with dopamine, and it may be possible to get rid of them with the help of the proposed technique, but it will not be possible to solve all the problems in this way.

Dr. Bechir Jarraya from the Department of neurosurgery at the Henri-Mondor Hospital in Paris, the lead author of the study, notes that the technique he proposed remains a therapy, not a panacea, because it only corrects the symptoms associated with dopamine, but does not cure the disease.

Over the past forty years, the so-called dopamine replacement therapy has been widely used. But since dopamine is delivered to the body discretely with injections, patients may develop involuntary movements. In this regard, scientists are looking for the possibility of a gradual accumulation of dopamine in the brain.

The results of the study are published by the journal Science Translational Medicine.

Prepared based on the materials of HealthDay: Gene Therapy Shows Promise for Parkinson's.

(It should be added that in an interview with HealthDay, Dr. Jarraya mentioned that clinical studies of this method of gene therapy for parkinsonism have already begun. Six volunteers have no serious adverse reactions, nor – at least, at the moment – encouraging results. But the study is not finished yet, and it will be possible to draw conclusions after receiving the final results – VM).

Portal "Eternal youth" http://vechnayamolodost.ru15.10.2009


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