Researchers from the University of Iowa working with Columbia University Medical Center ophthalmologists have corrected a blindness-causing gene mutation in stem cells derived from a patient using CRISPR/Cas9 gene editing technology.
Alexander Bassuk, MD, PhD, and Vinit Mahajan, MD, PhD, led a team of researchers who generated stem cells from patient skin cells and then repaired the damaged gene. The research was published in the journal Scientific Reports.
Here are five points:
1. The editing technique is so precise it corrected a single DNA change that had damaged the RPGR gene. More importantly, the corrected tissue had been derived from the patient’s own stem cells, and so could potentially be transplanted without the need for harmful drugs to prevent tissue rejection.
2. Dr. Mahajan said retinal diseases are a perfect model for stem cell therapy because medical experts have the advanced surgical techniques to implant cells exactly where they are needed.
3. The CRISPR technology was able to correct the RPGR mutation in 13 percent of the stem cells, which is a practically workable correction rate.
4. Developed three years ago, CRISPR/Cas9 has been heralded as a major breakthrough in genetic engineering, allowing scientists to easily, precisely and relatively cheaply make specific alterations to the DNA in cells and experimental animals.
5. The technology has not yet been used on humans, so safety concerns remain about the potential for unanticipated genetic changes, as well as ethical concerns given the technique’s capability to make permanent genetic changes in sperm and egg cells.
More articles on healthcare:
Become the ultimate customer — 5 key points on how freight management can drive down costs
30 things to know about ASC employee, physician compensation
Varied healthcare landscapes across the US
