Wednesday, February 3, 2016
Edited Stem Cells Offer Hope of Precision Therapy for Blindness (Doris Duke, Research to Prevent Blindness)
A team of University of Iowa and Columbia University Medical Center researchers have corrected a blindness-causing gene mutation in stem cells derived from an affected patient. The result offers hope that eye diseases might one day be treated with patients' own edited tissue.
With the aim of repairing deteriorating retinas in patients with the inherited blinding disease X-linked retinitis pigmentosa (XLRP), Alexander Bassuk and Vinit Mahajan led a team of researchers who generated stem cells from a patient's skin cells and then repaired the damaged gene, RPGR, using CRISPR/Cas9, a new gene-editing technology. The technique is so precise it corrected a single DNA change that had damaged the RPGR gene. And because the corrected tissue had been derived from the patient’s own cells, it could potentially be transplanted without the need for anti-rejection drugs.
The research was published Jan. 27 in the journal Scientific Reports. . .
The study was funded in part by grants from the National Institutes of Health, the Doris Duke Charitable Foundation, and Research to Prevent Blindness. . .