An ASU professor and his students have made a contribution toward finding the cause of Alzheimer’s disease by improving single-DNA base editing with an efficiency of up to 90% in human stem cells.
The results of the study, published in the journal "Stem Cell Reports," were directed by David Brafman, who is a Biological and Health Systems Engineering assistant professor, along with the assistance of several graduate students working in his lab, including Nicholas Brookhouser, a UA graduate student studying for a doctorate in clinical translational sciences.
According to the 2019 Alzheimer’s Disease Facts and Figures report conducted by the Alzheimer’s Association, around 140,000 Arizonans had Alzheimer’s disease in 2019.
The report also said Arizona has the highest projected rate of growth in the country with an estimation of 200,000 people having Alzheimer’s in Arizona by 2025.
Access to technology that can discover the cause of the disease is a step toward fixing the underlying disease itself.
According to Brafman, one of the main reasons the stem cell editing had a high success rate was a result of Brookhouser finding a way to work with clusters of regularly interspaced short palindromic repeats.
According to the National Institutes of Health, CRISPR is another technological approach to gene editing.
“You hear about how great CRISPR technology is, which is true, but then you don’t hear about the difficulty in implementing that tool,” Brafman said. “So what Nick did is he figured out a way to lower some of those bars to implementing tools.”
The study was funded by the NIH for ASU’s Brafman Lab. Elise Rabin, writer editor of the Division of Communication & Outreach at NIH, said via email that one of the goals of NIH is "building the research foundation that drives discovery.”
The improvement of CRISPR's success rate, which is often low due to its complexity, has emboldened the lab to take steps toward new studies, including figuring out the cause of Alzheimer’s disease.
“We plan on utilizing the technology in our lab to generate different models related to Alzheimer’s disease and different neurodegenerative diseases,” Brookhouser said.
The NIH also said gene editing is “a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed or altered at particular locations in the genome.”
Brafman said that the study of Alzheimer's disease is something the entire community should care about as it may become more prevalent in the future.
According to both Brookhouser and Brafman, one of the main takeaways from their research was the understanding that no one individual can make a breakthrough study on their own. Rather, it’s the culmination of a lot of efforts that can lead to a final result.
“Research isn't about curing the disease, the ultimate goal is making your contribution,” Brafman said.
Another highlight Brookhouser gleaned from the study was recognizing that success should be a regular occurrence throughout college, and students need to take advantage of the opportunities universities can offer them.
“I think that people can continue to take our work and improve it,” Brookhouser said. “It’s a good example of taking a current issue in our capability and making it more accessible to people.”
Brookhouser’s beliefs are emboldened not only by graduate students but by undergraduate students dedicated to the world of medical research as well.
“I mean, there’s so much potential regenerative capabilities ... here at ASU in general students have a sheer amount of opportunities,” said Jack Kollings, a sophomore double majoring in biological sciences and global health.
Kollings, who is also secretary of The Biomedical Science and Genetics Club at ASU, said he believes the study was not only a breakthrough in the field of stem cell research but paradigm in helping students comprehend that success doesn’t have to only come after college.
“This was a project composed by the students, and I was skeptical as faculty are often skeptical and they proved me wrong,” Brafman said. “And I’m happy to have been proven wrong.”