A team of researchers from Florida wants to develop a new screening tool for Parkinson's disease using the CRISPR Cas9 gene-editing technology, a crucial step to identifying new drug therapies for the degenerative disorder of the nervous system.
The technology allows scientists to assess and monitor a brain protein called alpha-synuclein that has been linked to Parkinson's disease, which affects motor functions caused by a gradual loss of brain cells.
"It's the most powerful and widely used gene-editing technique in use because it allows us to change the DNA in living cells," Associate Professor Yoon-Seong Kim said. "The innovation of this method is that it enables us to monitor this gene in real-time without killing the cell. Without the CRISP Cas-9 method, you would have to extract all the proteins from the cell to measure them, which kills the cell."
The team edited the alpha-synuclein gene using the CRISPR technique and inserted a luminescent tag made from proteins that allows fireflies to give off light. When the cell creates alpha-synuclein protein, the tag lights up.
"More light means an increased level of alpha-synuclein, which would be considered a diseased state," Levi Adams, one of the lead researchers, said.
"If we take one of these modified cells and treat it with a particular drug, if it doesn't produce light anymore, then this means the drug is a potential treatment for the disease," Sambuddha Basu, one of the researchers, said.
The easy and convenient light production process will allow researchers to perform high throughput screening and test a variety of drugs at once. Scientists also hope to identify other potential ways to reduce the production of alpha-synuclein, which could possibly prevent the development or progression of Parkinson's disease.
The findings of the study have been published in the journal Scientific Reports.