Scientists have used CRISPR-Cas9 to snip a genetic mutation that causes blindness out of stem cells derived from a patient. The research could hold potential for one day transplanting retinal tissue from a patient’s own stem cells to treat blindness. Here’s what lead researcher and ophthalmologist Dr. Vinit Mahajan of University of Iowa said about the findings, published this morning in Scientific Reports.
What did you do to edit out the gene mutation?
We took human stem cells from patients who have [an inherited form of] blinding eye disease. We used CRISPR-Cas9 to correct that mutation. But the region where patients get this mutation is … a difficult area to sequence or do gene editing. So this research is very exciting. For eye disease, surgical ability to transplant tissue is extraordinarily high. But it’s a question of how we get the best possible tissue or cells to transplant.
What’s the potential use for those cells?
We can try to turn those stem cells into retinal cells and then we could surgically transplant them. There are surgical stem cell transplants right now, but they depend on fetal stem cells, which come from another person. So [they] are at high risk of rejection from a patient’s immune system. But the assumption is the retinas might tolerate the patient’s own stem cells much better.