PhD student wins 2024 Illinois Innovation Award for work in ALS
Her research focuses on gene therapies using CRISPR-based technology
Alejandra Zeballos, a PhD student in bioengineering at the University of Illinois Urbana-Champaign, was awarded the 2024 Illinois Innovation Award for her work on next-generation therapies to advance the treatment of amyotrophic lateral sclerosis (ALS).
Zeballos, a member of the Gaj Lab, has been working on gene therapies for ALS that harness the power of CRISPR-based technology, a strategy used by bacteria to fight off viruses that has recently gained widespread interest as a research tool for gene editing.
The $20,000 Innovation Award was launched in 2007 to recognize University of Illinois Urbana-Champaign students whose research efforts can potentially have a significant impact on real-world problems.
Granted by the Technology Entrepreneur Center in The Grainger College of Engineering, the program has so far awarded a total of $430,000 to student researchers.
“I am incredibly grateful to receive the prestigious Illinois Innovation Award in recognition of my work. It is a true honor to be recognized among such an accomplished group of experts and innovators,” Zeballos said in a university press release.
Innovation Award winner designed system to target messenger RNA
The CRISPR-Cas9 system is a genome-editing tool inspired by a defense system that allows bacteria to remove viral DNA from their own genome, dampening the viral infection.
It consists of Cas9, a protein that can cleave the DNA, and a guide molecule that directs Cas9 to a specific target sequence. This system allows for precise gene editing, such as gene insertion, deletion, or modification, in a particular location of interest.
However, modifying the DNA can pose significant safety risks. The CRISPR-Cas9 system is not 100% accurate, and an accidental change in DNA can fundamentally alter cellular activity and may contribute to cancer.
To go around that, Zeballos designed a CRISPR system to instead target messenger RNA (mRNA) molecules. mRNAs are intermediate molecules that are produced when a gene is read and are used as templates for protein production. By modifying mRNA, the system can still introduce the modifications in the protein, but it does not interfere with the cell’s genome.
This recognition inspires me to strive for excellence and to continue to demonstrate that the University of Illinois Urbana-Champaign is a leading force in creating the next-generation gene therapies for neurologic diseases that will revolutionize traditional medicine.
In her research with the Gaj Lab — led by Thomas Gaj, PhD, an assistant professor in the department of bioengineering — Zeballos developed a CRISPR system that used the Cas13 protein and targeted the mRNA molecules that would be used as templates for the ataxin-2 protein.
The ataxin-2 protein has been identified as a potent modifier of the toxic TDP-43 protein clumps that are present in up to 97% of all ALS cases and are believed to act as important drivers of the disease. When the amount of ataxin-2 was reduced with the new system, the amount of TDP-43 clumps also was significantly reduced.
Along with other lab researchers, the PhD student also tested this CRISPR-based system in a mouse model of ALS. Data showed that it improved motor function and coordination, eased the loss of motor neurons, and extended survival in the mice.
Overall, these findings support the potential of CRISPR-based technologies for neurologic diseases, including ALS.
“This recognition inspires me to strive for excellence and to continue to demonstrate that the University of Illinois Urbana-Champaign is a leading force in creating the next-generation gene therapies for neurologic diseases that will revolutionize traditional medicine,” Zeballos said.
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