Researchers have devised a method of using gene therapy in rats to prevent Parkinson’s disease that they hope will translate to a new treatment for humans.
Parkinson’s disease is a neurological disorder that is characterized by the loss of the ability to control muscle movements. It occurs when nerve cells in a certain region of the brain deteriorate, leaving them no longer able to produce a vital brain chemical known as dopamine.
University of Pittsburgh researchers were aware that people with Parkinson’s disease have poorly functioning mitochondria, which produce energy in the body’s cells. The team was also aware that protein in the brain known as α-synuclein interacted with mitochondria in a damaging way, but these two areas of research had never been pursued together, the researchers said.
So the team decided to explore the connection further in rats. Using a harmless virus, they transported a small piece of genetic code into the nerve cells to block the production of the brain protein in rats. They then exposed the animals to the pesticide rotenone, which inhibits mitochondrial function.
They found that using the gene therapy prevented symptoms of Parkinson’s disease from appearing in the rats despite their exposure to the pesticide. Now they are hoping this discovery will lay the groundwork for clinical trials of the gene therapy, which they are hoping could ultimately lead to a new treatment for Parkinson’s disease, they said.
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