Synthetic DNA nanoparticles for gene therapy

(Press-News.org) CLEVELAND—Case Western Reserve University chemist Divita Mathur was awarded a National Science Foundation (NSF) Faculty Early Career Development Program (CAREER) grant for her research in synthetic DNA nanoparticles, which have potential applications in gene therapy.

The grant will support Mathur’s work in synthesizing nanoparticles and studying how they behave inside cells in a laboratory. She will use single-cell injections and a microscope to track the nanoparticles and watch what happens to them over time inside individual cells.

The CAREER award is considered the agency’s most prestigious grant to junior faculty members “who have the potential to serve as academic role models in research and education and to lead advances in the mission of their department or organization.” Mathur, assistant professor of chemistry in the College of Arts and Sciences, is among three CWRU faculty members awarded CAREER grants this year.

“This groundbreaking work is a great example of fundamental science that could lead to new life-saving treatments,” said David Gerdes, dean of the college. “The CAREER award shows that she is a rising star, not only on our campus, but also in our national scientific community.”

 

Mathur’s DNA nanoparticles are highly programmable and could be designed to encode a gene that replaces a missing or malfunctioning gene, instructing a cell to produce a needed protein or correct a genetic error.

In many genetic diseases, scientists know the gene that needs to be corrected, Mathur said.

“The problem is the delivery,” she said. “It’s easy to deliver things to the liver, so liver-based therapies are in clinical trials. Converting this to another area of the body is difficult, which is one of the things that motivates us to pursue this research.” 

Mathur said that eventually these DNA nanoparticles could also be designed with an attachment that functions like a barcode on an envelope, sending it to a particular address, or in this case, targeting a particular kind of cell.

The CAREER Award grant allows Mathur to delve into the basic question of how these manufactured DNA structures behave once they get inside a cell. To study that, she will attach a molecule to the DNA that fluoresces so the particles can be observed by microscope inside a living cell. 

“We don’t know how these nanoparticles act when they are inside the cell; how they interact with the proteins that are inside,” Mathur said. “We have to understand that fundamentally before we can imagine how to design therapeutics.”

The CAREER award also provides funding for Mathur to engage high school students in summertime chemistry research projects and for building mixed-reality, three-dimensional models of chemical and biochemical molecules.

“We want to show students how molecules are three-dimensional, how they occupy space and how they have a specific orientation in space, like right or left-handedness,” she said.

Earlier this year, Mathur received the university’s John S. Dieckhoff Award for Excellence in Graduate Mentoring. One of the undergraduates working in her lab, Sara Desai, received the prestigious Barry Goldwater scholarship, a highly competitive national scholarship that provides tuition assistance for students who intend to pursue research careers in science, mathematics or engineering.

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