Gene Therapy & Possible Treatments For Parkinson's & Alzheimer's Patients

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.

The cause of Alzheimer's disease, which is a type of dementia, is still a subject of serious research. There are many hypotheses that are currently being looked at to determine the causes of this disease in which the synapses and neurons of the cerebral cortex begin to die off. In addition, amyloid plaque or excesses of proteins have been found in the brains of people who have Alzheimer's disease.

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.

Gene therapy, which is a type of therapy in which DNA is basically used as medicine or pharmaceutical agent, is being looked at as a possible way to slow or stop the effects of neurodegenerative diseases. In gene therapy, scientists introduce a non-infectious virus into the host, which moves a gene into the brain. This gene then produces a specific enzyme that will either protect the host's brain from further damage or help reduce the severity of symptoms.

There have been several studies of using gene therapy to treat Parkinson's disease, and a few of these studies have yielded positive results. A 2011 study published in Lancet Neurology, for example, looked at the results of gene therapy in a double-blind trial. It was found that those people who received the gene therapy had a strong improvement in motor control compared with those who did not receive the therapy. This is just one of several gene therapy trials that have been successful in the treatment of Parkinson's.

The studies regarding Alzheimer's disease have been less successful; however, a 2011 study did show the reduction of the amyloid plaque when gene therapy was applied. The subjects in this study were mice who had been given an inactive HIV lentivirus, which causes the plaque to form much as it does in a patient with Alzheimer's. While these types of studies do not necessarily provide the hope a cure for the disease, they certainly suggest that it might soon be possible to slow or stop the progression of this debilitating condition.




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