A team of researchers is using breakthrough gene-editing technology to develop a new screening tool for Parkinson’s disease. The technology allows scientists in the lab to “light up” and then monitor alpha-synuclein, a brain protein that has been associated with Parkinson’s. The study was published in Scientific Reports.

The researchers used CRISPR Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) gene-editing technology. The system is one of research’s fastest growing biomedical techniques that allows scientists to make specific changes in the DNA of plants and animals while not killing cells. The system is becoming instrumental in studying genetically based treatments for diseases including cancer and Parkinson’s.

Using the CRISPR technique, the team edited the alpha-synuclein gene and inserted a luminescent tag made from the same proteins that cause fireflies to light up. Every time the cell creates the alpha-synuclein protein, the tag gives off a light. The team found that measuring the light was a reliable method to measure alpha-synuclein production.

With the engineered cells, researchers can screen new and existing drugs to see how they regulate alpha-synuclein level in patients. The scientists hope to go on to identify ways to reduce alpha-synuclein production that can possibly prevent Parkinson’s or its progression in patients diagnosed with the disease.

Paper: “A novel tool for monitoring endogenous alpha-synuclein transcription by NanoLuciferase tag insertion at the 3′end using CRISPR-Cas9 genome editing technique”
Reprinted from materials provided by University of Central Florida.

September 1, 2017