Patient advocacy organizations should consider the potential CRISPR gene-editing technology has to help fix the most dangerous genetic mutations that have no cure, Debra Miller, CEO and founder of CureDuchenne, an advocacy organization dedicated to research on Duchenne muscular dystrophy, wrote in STAT.
"As the mother of a son with Duchenne muscular dystrophy, a cruel genetic disease that breaks down muscle until the body can no longer function, I don't see CRISPR as scary or dangerous," Ms. Miller wrote. "To me, it represents the best hope we have for curing my son's disease and possibly many others."
CureDuchenne provided seed funding for a company founded by Dallas-based UT Southwestern Medical Center professor Eric Olson, PhD. The company, Exonics Therapeutics, is the first dedicated to finding a cure for Duchenne through CRISPR.
In a preclinical study last year, Dr. Olson and his research team restored 92 percent of dystrophin — a protein that helps keep muscle cells intact and that Duchenne depletes — in animal hearts. The findings inspired hope for human trials soon. The CRISPR-based treatment is delivered to the tissues of patients with Duchenne.
"We need to ask ourselves what CRISPR should be used for. I think of it in much the same way that I think of an eraser on a pencil: correcting genes like correcting misspelled words on a page," Ms. Miller wrote. "We should use this technology to fix the most dangerous mutations that have no cure. It could someday be applied to the 10,000 or so genetic diseases that afflict 75 million people around the globe, or even to chronic diseases like cancer and diabetes that affect billions of people."