A single change in a protein may play a role in whether someone develops Parkinson's disease, say University of Florida Genetics Institute researchers writing in a recent issue of the Proceedings of the National Academy of Sciences.
Scientists studying rats induced to display a form of Parkinson's disease discovered that a protein commonly found in brain cells can be toxic if - at one pinpoint location in its amino acid structure - it lacks a chemical compound called a phosphate.
When scientists used gene therapy to simulate a phosphate at this critical position, the rats' brain cells didn't develop the Parkinson-like pathology that would normally occur.
The finding provides new insight into the fundamentals of Parkinson's disease and the role of an abundant yet mysterious brain protein known as alpha-synuclein, which is believed to help brain cells communicate but may have a more sinister role in the development of neurological diseases.
“We have another potential target for therapy, but there is a great deal left to discover,” said Nicholas Muzyczka, Ph.D., a professor of molecular genetics and microbiology in the College of Medicine and an eminent scholar with the UF Genetics Institute. “This is one more piece of information about what might be causing the toxicity in Parkinson's disease, and it gives us a little more to go on about what alpha-synuclein does in the brain.”
Generally located at the synapses of nerve cells, alpha-synuclein is believed to aid in brain function, possibly by helping cells communicate with one another by controlling the release of neurotransmitters such as dopamine.
Mutations of alpha-synuclein may cause a rare, inherited form of Parkinson's, and the protein has been found to be the major component of Lewy bodies, which are abnormal clusters of protein in the brain cells of patients with Parkinson's disease.
The National Parkinson Foundation estimates 1.5 million Americans currently have Parkinson's disease and about 60,000 new cases are diagnosed each year. It is caused by the death or impairment of certain nerve cells in a part of the brain called the substantia nigra. When these cells die, the body is deprived of dopamine, a neurotransmitter vital for movement.