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On the Fulfilling the Promise of Genetic Surgery Treatment Frontier: By Michael Ferguson semi-random," says Osborn, who is also a member of the University of Minnesota's Center for Genome Engineering. "They can disrupt other important sequences, and some gene therapy trials have actually led to more severe disease from the treatment itself because the genetic landscape has been disrupted." Those experiences drove researchers to develop more precise methods for delivering proteins to targeted DNA. "People started to generate these proteins that could be designed for particular spots in the genome, [and then] co-localize in that spot and cut the DNA," Osborn explains. "In response to that, the cell actually up- regulates an error-free repair pathway called homologous recombination." Researchers must custom-build these proteins, and in the past 20 years, significant progress has been made to refine delivery systems and the speed with which they can be designed. Since he entered the field, Osborn notes, the protein-building process has been reduced from one year to less than one week. "There's been a change in the platform, but really the speed and availability of these reagents have opened the door to many people who otherwise wouldn't be able to employ [them]," Osborn says. "They've certainly become more user-friendly." T HE HUMAN GENOME is composed of more than 3 billion base pairs of DNA, and alterations to a single base can cause a broad range of catastrophic diseases, such as Down syndrome and sickle cell anemia, as well as more obscure but similarly devastating conditions, such as epidermolysis bullosa (EB). Seeking curative treatments for such disorders, researchers have developed several methods for editing faulty genes using genetic surgery. Genetic surgery allows physicians to resect faulty portions of DNA bases and replace them with corrected functional cop- ies. However, directing corrected genes to the precise DNA proved an early challenge for researchers, according to Mark Osborn, PhD, Assistant Professor in the Division of Blood and Marrow Transplantation at the University of Minnesota Medical School. "Those genes have to go somewhere in the genome, and where they go is A RAPIDLY DEVELOPING APPROACH MAY REWRITE THE BOOK ON HOW TO TREAT GENETIC DISORDERS. 2 8 | Minnesota MD NEWS ■ M D N E W S . CO M