Pictured: UCF scientists. Credit: UCF, College of Medicine
We’ve spoken a lot about the potential uses of CRISPR – the gene-editing technology that has been a constant of health research headlines recently.
The technique, which allows scientists to make specific changes in DNA without killing cells, has now been harnessed in what is being labelled a ‘breakthrough’ in the diagnosis and treatment of Parkinson’s disease.
A team of researchers at the University of Central Florida has used the technology to “light up” and monitor a brain protein called alpha-synuclein that has been associated with Parkinson’s.
Everyone has alpha-synuclein present in their brain but a person with Parkinson’s will develop abnormal levels of the protein. The researchers’ hope is that by monitoring the protein in the cell they will be able to measure what causes it to go up and what treatments can make it go down. This could be beneficial in the identification of new drug therapies for Parkinson’s disease.
Parkinson’s disease is a debilitating degenerative disorder involving the malfunction and death of vital nerve cells in the brain, called neurons. The disorder affects around 80,000 Australians. There is currently no single test to diagnose it and no cure.
The team used CRISPR to edit the alpha-synuclein gene and inserted a luminescent tag made from proteins that light up. This meant that every time a cell created the alpha-synuclein protein, the tag would give off a light, making it much easier to monitor if too much alpha-synuclein was being produced.
Associate Professor Yoon-Seong Kim, one of the study’s lead researchers said;
“CRISPR is the most powerful and widely used gene-editing technique in use because it allows us to change the DNA in living cells.
The innovation of this method is that it enables us to monitor this gene in real-time without killing the cell. Without the CRISPR Cas-9 method, you would have to extract all the proteins from the cell to measure them, which kills the cell.”
With the new technology, the scientists hope to identify ways to reduce alpha-synuclein production that can possibly prevent Parkinson’s or its progression in patients diagnosed with the disease.
“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 this disease,” Sambuddha Basu, another researcher on the team, said.
With the engineered cells, the researchers will screen new and existing drugs and hope to also focus on what aspects of the alpha- synuclein protein kill neurons during Parkinson’s disease.
The team published its findings in the Scientific Reports journal.
