Researchers and drug companies are enthusiastic about the use of genome editing tools like CRISPR-Cas9 to replace disease-causing genetic variants with healthy DNA stretches, the Wall Street Journal reports.
It cautions that therapies based on such approaches won't be available for years, if at all, but adds that they offer "tremendous potential for new treatments."
"What if you could go right to the root cause of that disease and repair the broken gene? That's what people are excited about," Katrine Bosley, chief executive of Editas Medicine, says.
Her company, the Journal notes, is pursuing a CRISPR-Cas9-based therapeutic for Leber congenital amaurosis, while others are investigating its use to treat sickle-cell anemia, beta thalassemia, and blood cancers.
There are, though, a few stumbling blocks. There's a patent fight hanging over the field that involves the Broad Institute, the University of California, Umea University, and others regarding which team — Feng Zhang at the Broad or Berkeley's Jennifer Doudna and Umea's Emmanuelle Charpentier — first developed the approach.
There's also the question of how to get any CRISPR-Cas9-based therapy to its intended site. The first treatments will likely involve taking a sample of a patient's blood, treating the blood cells, and returning them, the Journal says, as other routes like using adeno-associated viruses or lipid nanoparticles are explored.
"In 10, 15 years, our relationship with genetic disease will be very different from today," Jacob Corn, managing director of the Innovative Genomics Initiative, tells the Journal. "It will be, 'Oh, my child was born with sickle cell. We're just going to change that.'"