mardi 10 septembre 2013

Gene Therapy For Alzheimer's & Parkinson's Disease

By Armand Zeiders


Neurodegenerative diseases affect millions of people worldwide. In the United States alone, there are more than 5 million Americans living with Alzheimer's disease, and another million Americans have been diagnosed with Parkinson's disease. These diseases cause the death of neurons in the brain leading to a host of serious neurological and biological issues.

Alzheimer's disease is a specific form of dementia, and although the actual cause of the disease remains unknown, scientists currently are studying all aspects of this disease. The disorder affects the cerebral cortex and its synapses and neurons, which begin to die as the disease progresses. The brains of those affected by Alzheimer's also have been found to contain excessive amounts of proteins in the form of amyloid plaque.

The causes of Parkinson's disease are equally difficult to determine, but in general, scientists can point to gene mutations as the cause of these neurodegenerative disorders. In Parkinson's disease, it is the central nervous system which is affected by the death of cells that produce dopamine. The symptoms of Parkinson's include everything from uncontrollable tremors, muscular stiffness, balance problems, speech problems and even slower cognitive functioning.

Scientists are currently studying and working on using gene therapy to slow, stop or cure these types of diseases. In this process, DNA is used as a form of medicine and added directly into cells using a non-infectious type of virus. The gene then enters the cell and begins producing enzymes that are meant to stop or slow the growth or progression of the disorder.

There are many different groups of scientists studying gene therapy and Parkinson's disease, and a few of these studies have yielded encouraging results. Lancet Neurology, for example, posted a study from 2011 in which scientists completed a double-blind trial with 45 Parkinson's patients. The group that actually received the therapy saw a much higher improvement in their motor control, while those that did not receive it had only a 12 percent improvement on average. There are several more examples of successful gene therapy studies, which hold great promise of eventually finding a way to slow or stop the disorder entirely.

Thus far, gene therapy studies in regards to treating Alzheimer's disease have not been as successful. A recent study in which mice were given gene therapy after being given an inactive type of an HIV virus showed positive results. The particular HIV lentivirus given to these mice caused a similar growth in amyloid plaque as found in patients with Alzheimer's. The gene therapy with the mice was found to reduce the amount of plaque, suggesting that the plaque might also be reduced in Alzheimer's patients when treated with the same type of gene therapy.




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