ChristianaCare: Advancing Gene Editing Through In-Vitro Approach
Even after 20 years of leading research into gene-editing techniques and applications, ChristianaCare’s Gene Editing Institute continues to advance the science.
Examples include recently pioneering a new technique to better evaluate unintentional damage done during gene repair, developing a potential pathway to allow chemotherapy in late-stage lung cancer patients and devising an educational kit to better explain and teach gene therapy to students.
Much of this work is being led by the institute’s largely younger, female staff of researchers. For example, Brett Sansbury has not even finished her doctorate at the University of Delaware, and her undergraduate degree is in biological chemistry, with a specialization in agrochemical precursors — not the standard background for a researcher in gene editing. Yet she is lead author in two recent journal articles explaining the advances in CRISPR gene editing being made by her and her colleagues at the institute.
CRISPR (clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found within the genomes of prokaryotic organisms such as bacteria and archaea and is becoming a primary tool in gene therapy.
Sansbury’s work using a novel approach to in-vitro gene editing has resulted in a methodology that better identifies unintended and potentially harmful changes or mutations during CRISPR editing. It may serve as a platform that simplifies the theoretical and hands-on educational tools used to teach genetics to even high school students.
“We were the first to approach CRISPR in quite this manner,” says Eric Kmiec, director of the Gene Editing Institute. “Brett was the right person to do this work. There were not a lot of quantitative studies in the past; rather, it has been more about phenomenon.”
“I’ve always liked solving problems,” Sansbury laughs in explaining her approach to research. “Math is my friend, and chemistry is my friend.”
It is also an approach that Kmiec says characterizes the 22-person institute, most of whose staffers are researchers, not practicing physicians, and most of whom “are young and female. They don’t settle for an answer. They want the right answer.”
Another institute program is seeking to treat patients with late-stage non-small-cell lung cancer, testing whether using CRISPR to disable a particular gene would allow standard chemotherapy to work better and longer. Clinical trials for the treatment will begin soon, using newly formulated FDA guidelines.
“This would not be a cure for late-stage lung cancer,” Kmiec emphasizes, “but it could hopefully extend [patients’] lives by a few months” that might allow them to participate in important family events. The therapy might eventually be applied to pancreatic cancers as well.
Finally, another colleague, Natalia Rivera-Torres, has translated Sansbury’s work into an educational kit, which will be marketed shortly, to train the next generation of genetic scientists.
The kit was developed with educational input from faculty and students at Delaware Technical Community College using a $1 million National Science Foundation grant.