Biomedical Engineering Professor Receives NIH Award for Research in Genome Engineering

Photo Submitted

Christopher Nelson, assistant professor of biomedical engineering.

The National Institutes of Health awarded a University of Arkansas biomedical engineering faculty member a three-year, $735,000 award for research in genome engineering.

Christopher Nelson, assistant professor of biomedical engineering, also holds the 21st Century Professorship in Biomedical Engineering. He has worked for five years on genome engineering approaches for genetic diseases. Nelson's previous research focused on Duchenne muscular dystrophy, a severe genetic disease. Patients with the condition have a life expectancy of 20 to 30 years.

Genome engineering is an alternative treatment to gene therapy. While gene therapy relies on the delivery of external DNA, CRISPR permanently modifies the host genome directly. Because the effects of gene therapy fade overtime because of inevitable cell division, genome engineering is a more permanent solution, as the altered DNA is copied and transferred to the new cell.

Nelson's research, transitioned from his postdoctoral program at Duke University, has proven the longevity of genome engineering. Mice who suffered from Duchenne muscular dystrophy maintained edited DNA over their lifespans. The success of this research earned his lab their first NIH award, a three-year grant that will allow the expansion of genome engineering methods to other genetic diseases.

Nelson and his lab aim to focus first on Hemophilia, a genetic disease that affects the blood's ability to clot. Nelson's lab also intends to study the potential side effects of genome editing, as well as reduce the immune system's response, or immunogenicity, to the genome editing process, which currently alerts the immune system to attack the altered cell proteins.

"The immune system may be a barrier to genome engineering's efficiency in humans," Nelson said. "Overcoming this barrier will allow genome editing to be successful in the clinic."

"I am extremely excited Dr. Nelson has received this highly competitive NIH-R00 award as a follow-up to the NIH-K99 postdoctoral research award at Duke University," said Raj Rao, head of the Department of Biomedical Engineering. "The work Dr. Nelson will conduct combines new technologies in biomaterials and genome engineering and provides a new disciplinary focus for the department. His research over the next three years will allow graduate students to venture into biomaterials and gene delivery work and ultimately open new collaborative avenues for biomedical research on campus."