The Use Of Gene Therapy To Treat Parkinson’s Disease And Alzheimer’s

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.
Parkinson’s causes are equally mystifying, but the common link between these two neurodegenerative diseases is that both are cause by gene mutations. How these genes become mutated is unknown. Where Alzheimer’s causes severe dementia, Parkinson’s attacks the dopamine in the cells of the central nervous system causing the trademark tremors and stiff gait you often see in Parkinson’s patients. In addition, there are balance and speech problems and often a loss in cognitive function.
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 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.
Researchers so far have had less success using gene therapy to combat Alzheimer’s disease, although a 2011 study showcased the reduction of the amyloid plaque after therapy was used. This study was completed using mice that had been injected with an inactive type of the HIV lentivirus. This particular virus cause amyloid plaque to build in much the same way as the plaque builds in Alzheimer’s disease. This type of study is an excellent first step into eventually being able to use some form of gene therapy to stop or slow this disease.
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