Nav: Home

CRISPR tech leads to new screening tool for Parkinson's disease

June 05, 2017

A team of researchers at the University of Central Florida is using breakthrough gene-editing technology to develop a new screening tool for Parkinson's disease, a debilitating degenerative disorder of the nervous system. The technology allows scientists in the lab to "light up" and then monitor a brain protein called alpha-synuclein that has been associated with Parkinson's.

"Alpha-synuclein is a protein that is normally found in the brain. We all have it," said Burnett School of Biomedical Sciences doctoral student Levi Adams, one of the lead researchers on the project. "But for some reason, when you have Parkinson's the levels become abnormal. So if we can monitor this protein in the cell, we can start to measure what causes it to go up and also what treatments can cause it to go down."

The team published its findings in the Scientific Reports journal. The National Institutes of Health (5R21NS088923-02 ) funded the work. The researchers believe their work is a crucial step toward identifying new drug therapies for Parkinson's disease.

Adams is partnering with doctoral student Sambuddha Basu, associate professor and neurosciences researcher, Associate Professor Yoon-Seong Kim, and scientist Subhrangshu Guhathakurta to study Parkinson's, which affects motor functions caused by a gradual loss of brain cells. There are about 60,000 new cases of Parkinson's each year in the United States.

They are using 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.

"It's the most powerful and widely used gene-editing technique in use because it allows us to change the DNA in living cells," said Kim, who is also a medical doctor. "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."

Using the CRISPR technique, the Burnett team edited the alpha-synuclein gene and inserted a luminescent tag made from proteins that causes fireflies to light up. Every time the cell creates the alpha-synuclein protein, the tag gives off a light. That reaction "makes it much easier to measure," Adams said. "More light means an increased level of alpha-synuclein, which would be considered a diseased state."

The team found that measuring light was a reliable method to measure alpha-synuclein production.

"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," Basu said.

With the engineered cells, researchers can screen new and existing drugs to see how they regulate alpha-synuclein level in patients.

"With an easy-to-measure reporter like light production, this will allow us to do high throughput screening, where you can test a large panel of drugs at once," Guhathakurta said.

With the new technology, the scientists hope to identifying ways to reduce alpha-synuclein production that can possibly prevent Parkinson's or its progression in patients diagnosed with the disease.

The team said research will focus on what aspects of the alpha- synuclein protein kill neurons during Parkinson's disease.
-end-
The University of Central Florida, one of the largest universities in the nation with more than 64,000 students, uses the power of scale and the pursuit of excellence to make a better future for our students and society. Described by The Washington Post as demolishing "the popular belief that exclusivity is a virtue in higher education" and credited by Politico with creating a "seamless pipeline of social mobility," UCF is recognized as one of the best values in higher education. UCF aligns its teaching, research and service with the needs of the community and beyond, offering more than 200 degree programs at more than a dozen locations, including its main campus in Orlando. Faculty and students are creating innovations in areas as diverse as simulation and training, optics and lasers, hospitality management, video game design, business, education and health care to solve local and global problems. For more information, visit http://www.ucf.edu.

University of Central Florida

Related Brain Articles:

Unique insight into development of the human brain: Model of the early embryonic brain
Stem cell researchers from the University of Copenhagen have designed a model of an early embryonic brain.
An optical brain-to-brain interface supports information exchange for locomotion control
Chinese researchers established an optical BtBI that supports rapid information transmission for precise locomotion control, thus providing a proof-of-principle demonstration of fast BtBI for real-time behavioral control.
Transplanting human nerve cells into a mouse brain reveals how they wire into brain circuits
A team of researchers led by Pierre Vanderhaeghen and Vincent Bonin (VIB-KU Leuven, Université libre de Bruxelles and NERF) showed how human nerve cells can develop at their own pace, and form highly precise connections with the surrounding mouse brain cells.
Brain scans reveal how the human brain compensates when one hemisphere is removed
Researchers studying six adults who had one of their brain hemispheres removed during childhood to reduce epileptic seizures found that the remaining half of the brain formed unusually strong connections between different functional brain networks, which potentially help the body to function as if the brain were intact.
Alcohol byproduct contributes to brain chemistry changes in specific brain regions
Study of mouse models provides clear implications for new targets to treat alcohol use disorder and fetal alcohol syndrome.
Scientists predict the areas of the brain to stimulate transitions between different brain states
Using a computer model of the brain, Gustavo Deco, director of the Center for Brain and Cognition, and Josephine Cruzat, a member of his team, together with a group of international collaborators, have developed an innovative method published in Proceedings of the National Academy of Sciences on Sept.
BRAIN Initiative tool may transform how scientists study brain structure and function
Researchers have developed a high-tech support system that can keep a large mammalian brain from rapidly decomposing in the hours after death, enabling study of certain molecular and cellular functions.
Wiring diagram of the brain provides a clearer picture of brain scan data
In a study published today in the journal BRAIN, neuroscientists led by Michael D.
Blue Brain Project releases first-ever digital 3D brain cell atlas
The Blue Brain Cell Atlas is like ''going from hand-drawn maps to Google Earth'' -- providing previously unavailable information on major cell types, numbers and positions in all 737 brain regions.
Landmark study reveals no benefit to costly and risky brain cooling after brain injury
A landmark study, led by Monash University researchers, has definitively found that the practice of cooling the body and brain in patients who have recently received a severe traumatic brain injury, has no impact on the patient's long-term outcome.
More Brain News and Brain Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Listen Again: Meditations on Loneliness
Original broadcast date: April 24, 2020. We're a social species now living in isolation. But loneliness was a problem well before this era of social distancing. This hour, TED speakers explore how we can live and make peace with loneliness. Guests on the show include author and illustrator Jonny Sun, psychologist Susan Pinker, architect Grace Kim, and writer Suleika Jaouad.
Now Playing: Science for the People

#565 The Great Wide Indoors
We're all spending a bit more time indoors this summer than we probably figured. But did you ever stop to think about why the places we live and work as designed the way they are? And how they could be designed better? We're talking with Emily Anthes about her new book "The Great Indoors: The Surprising Science of how Buildings Shape our Behavior, Health and Happiness".
Now Playing: Radiolab

The Third. A TED Talk.
Jad gives a TED talk about his life as a journalist and how Radiolab has evolved over the years. Here's how TED described it:How do you end a story? Host of Radiolab Jad Abumrad tells how his search for an answer led him home to the mountains of Tennessee, where he met an unexpected teacher: Dolly Parton.Jad Nicholas Abumrad is a Lebanese-American radio host, composer and producer. He is the founder of the syndicated public radio program Radiolab, which is broadcast on over 600 radio stations nationwide and is downloaded more than 120 million times a year as a podcast. He also created More Perfect, a podcast that tells the stories behind the Supreme Court's most famous decisions. And most recently, Dolly Parton's America, a nine-episode podcast exploring the life and times of the iconic country music star. Abumrad has received three Peabody Awards and was named a MacArthur Fellow in 2011.