Bioengineers are using a new gene-editing system and glow-in-the-dark sea creatures to advance the fight against Parkinson’s disease.
Parkinson’s disease is a degenerative disease that causes tremors, muscle rigidity and restricted mobility. There is no cure, but the search is on for new drugs that can halt the disease in its tracks.
Bioengineers in central Florida are the first in the U.S. to use a new, cutting-edge technique that may pave the way for very early stage treatments.
A million Americans are living with Parkinson’s, and for many, current medications don’t always control the side effects and tremors.
“They’ve affected me since I was in my 30s," Harriet Marksfield said.
“Sometimes, you get so overwhelmed as far as being tired," Helen Michaelson said. "You just can’t go anymore
Researchers at the University of Central Florida are using new technology to develop a screening tool. A gene-editing system called CRISPR allows them to “light up” a protein in the brain associated with Parkinson's.
“In this study, we are working with a protein that was actually isolated from nature," doctoral student Levi Adams said. "It’s very similar to something you can find in a firefly, when fireflies light up
In this case, scientists used deep-sea shrimp, which also have the same ability to glow in the dark. Using the CRISPR system, scientists inserted the deep-sea shrimp gene into human cells in the lab so they could more easily measure changes in the Parkinson’s protein.
“The expression of this protein goes up in cases of Parkinson’s disease, which leads to the subsequent, degenerative process,” Dr. Sambuddha Basu said.
Using the engineered cells, scientists can also screen new drugs to see if they lower the protein level in patients.
“This is the first step in allowing us to identify potential treatments for this disease,” Adams said.
The CRISPR system allows researchers to change the DNA of plants and animals without killing cells, and with faster results. CRISPR is being used more frequently in the study of genetic treatments for diseases like cancer and Parkinson’s.
RESEARCH SUMMARY
REPORT: MB #4393
CRISPR TECHNOLOGY: CRISPR technology is a simple, powerful tool used to edit genomes. This allows researchers to easily modify gene function by altering DNA sequences. It has many potential applications, some include; preventing the spread of diseases, treating diseases, improving crops, and correcting genetic defects. However it also raises ethical concerns. The technology was adapted from the natural defense mechanisms of archaea or the domain of single-celled microorganisms and bacteria. These organisms use CRISPR RNA and various proteins to stop attacks from viruses and other foreign bodies, primarily chopping up and destroying the DNA of the foreign invader. When these components are transferred in to more complex organisms, it allows for the manipulation or editing of genes.
(Source: https://www.livescience.com/58790-crispr-explained.html)
ALPHA-SYNUCLEIN: Alpha-synuclein is a protein that’s function in a healthy brain is currently still unknown. However, it is of great interest to Parkinson’s researchers because it is a major component of Lewy bodies. Lewy bodies are protein clumps that are a pathological staple of Parkinson’s disease. In the years since its discovery, the focus of researchers has been working to characterize the protein’s role in Parkinson’s and its potential target for neuroprotective therapies. There are still more questions than answers at this point as to whether or not and exactly how alpha-synuclein plays a direct causative role in Parkinson’s disease.
(Source: https://www.michaeljfox.org/understanding-parkinsons/living-with-pd/topic.php?alpha-synuclein)
FIREFLIES FOR RESEARCH: A research lab is monitoring and modulating the levels of alpha-synuclein so they do not reach a high level of toxicity associated with Parkinson’s disease. To do this, they need a tool to screen for drugs that can actually lower the levels of alpha-synuclein in human patients. They modeled the alpha-synuclein and tagged it using the CRISPR technique with a luminescent light-emitting gene called Nanoluc or Nanoluciferase found in fireflies. This ensured that whenever the protein is produced in a cell it will glow and emit light. The ultimate target of their research is to work on reducing the light that has been produced, as this will mean they have found drugs that help lower the expression of this alpha-synuclein protein that is associated with Parkinson’s disease.
(Source: Sambuddha Basu, PhD)
http://www.wndu.com/content/news/Fighting-Parkinsons-with-deep-sea-shrimp-and-CRISPR-478329963.html
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