WILMINGTON, Del. — Someday, a host of genetic diseases – ranging from sickle cell anemia to muscular dystrophy – could be curable using a technique that stops the disorders where they start: in a patient’s DNA.

What sounded like fantasy only a decade ago is now close to becoming a reality, thanks to newly discovered tools in the field of gene editing.

Millions of dollars are being poured into research and development of new therapies based on work now underway at some of the nation’s leading universities and cutting-edge startups.

Doctors at Christiana Care’s Helen F. Graham Cancer Center & Research Institute believe the rapidly growing field also holds promise for fighting leukemia, lymphoma and even liver cancer.

Through the newly formed Center for Translational Cancer Research, the hospital has teamed up with longtime gene editing researcher Eric Kmiec in the hopes of using the new science to find personalized cures for widespread diseases.

This spring, they formed the Gene Editing Institute, a partnership that seeks to combine research and drug development with laboratory services and education.

“The Gene Editing Institute places our translational science program on equal footing with the very best in the nation, perhaps even the world,” said Dr. Nicholas Petrelli, the medical director of the Graham Cancer Center. “This area is exploding so everyone wants to do it, but right now this is the only program in the tri-state area.”

Gene editing is essentially a three-step technique that first hones in on a mutated or abnormal section of DNA that’s causing a disease and then uses nucleases, or molecular scissors, to snip out the culprit before deleting, modifying or replacing it with a strand of normally functioning chromosome.

Kmiec, a molecular geneticist who’s been working in the field for nearly 30 years, compares the process to using spell check on a computer.

“If a word is spelled incorrectly – a letter is wrong – spell check can find it, compare it to a database and make corrections to that precise error while leaving the rest of the word alone,” he said. “That’s basically what we’re doing here, except the word in this case are the letters that make up a strand of DNA.”

The concept of gene editing has been around for decades, but the ability to effectively carry out the process has advanced in fits and starts, as scientists explore methods for conducting each step.

A video provided by Christiana Care shows how the hospital is teaming up with longtime gene editing researcher Eric Kmiec in the hopes of using the new science to find personalized cure for widespread diseases. 10/11/15
Courtesy of Christiana Care

Identifying the culprit section of DNA, developing tools to target that exact area and then making “edits” on a viable scale each have posed their own challenges.

“It’s always been hard, for instance, to regulate where and when the process takes place,” Kmiec said. “We’ve been able to do it on a limited scale for years, but you might get one in 100 cells that would change, so the frequency was not really clinically applicable.”

In the last decade, however, new designer proteins genetically engineered from plants and bacteria have been developed that now allow researchers to more efficiently conduct microsurgery on genes.

One such protein called CRISPR-Cas9 – an enzyme that bacteria use to fight viruses – has been hailed as the biggest scientific breakthrough of the century and is widely expected to be in contention for a future Nobel Prize.

The patent rights for the enzyme is being hotly contested, even as various research groups race to become the first to use the tool in therapeutic trials on humans.

With funding from the National Institutes of Health, Kmiec and his team of post-doctoral researchers, meanwhile, are working to develop the next generation of molecular scissors based on his prior research with sickle cell anemia at University of Delaware and Delaware State University.

The institute already has joined the Gene Editing Research Groups of the Association of Biomolecular Research Facilities, a consortium that includes the Massachusetts Institute of Technology, Stanford University and the Stowers Institute for Medical Research.

“We’re in a good position as a lab because we’ve been through a lot of the early work, understanding the regulation and mechanism of how the whole process works,” said Kmiec, who previously led UD’s Biotechnology Institute and Marshal University’s Institute for Interdisciplinary Research.

“That’s why coming to Christiana was a great opportunity because now we can leverage our background and knowledge of the field and apply it to what Dr. Petrelli has set up here,” he said.

The Center for Translational Cancer Research was launched in 2010 as a collaboration between Christiana Care, A.I. duPont Children’s Hospital and the Delaware Biotechnology Institute.

The idea, Petrelli said, is to identify new cancer treatments by encouraging cooperation among scientists, clinicians and biotech companies, like Genome Profiling, or GenPro, a startup that is developing tests to identify the early gene mutations that lead to cancer and other diseases.

Kmiec’s team hopes to eventually use gene editing techniques to cut and paste DNA mutations from existing cancer patients into stable cell lines that can be licensed to pharmaceutical companies, allowing them to safely test early-stage drug discoveries that target cells through the sequence of mutations that lead to unchecked tumor growth.

At the same time, the Gene Editing Institute is working to train scientists in how to use the latest techniques, while also developing new tools for teaching future genomic engineers.

The institute is working with the California-based life science company Bio-Rad to develop classroom kits that college and high school students can use to conduct gene editing experiments.

“We want to engage people in the science so they can go out and develop their own projects,” Kmiec said. “Everybody wants gene editing to work therapeutically and we think it will, but our goal here is also to make sure we advance the science in the right way.”

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