'The first therapeutic use of CRISPR will be to treat an eye disease'

A cross-country team of investigators at Columbia University Medical Center and the University of Iowa has been doing some preclinical feasibility research into using CRISPR gene editing technology to develop a personalized cure for retinitis pigmentosa, a condition that afflicts some 1.5 million people and eventually leads to blindness.

The team of scientists created stem cells from the skin of patients with the disease, then used CRISPR to correct the mutated RPGR gene that spurs retinitis pigmentosa. CRISPR/Cas9 tech has been swiftly adopted by academic research groups around the world. The tech offers a route to cut and splice DNA, a radical advancement that has spawned a variety of biotech startups.

But this particular team says that their work highlights how the human eye makes the ideal initial target for CRISPR R&D. The eye is easily accessed and monitored and scientists have plenty of past experience to prove that it can accept new tissue. By using cells from patients, they add, the new science can further up the odds of avoiding any rejection of inserted material.

"The X-linked form of retinitis pigmentosa is an ideal candidate for a precision medicine approach because a common mutation accounts for 90% cases," says Dr. Stephen Tsang, the László Z. Bitó Associate Professor of Ophthalmology and associate professor of Pathology & Cell Biology at CUMC, and one of the study's senior authors.

"Our vision is to develop a personalized approach to treating eye disease," Tsang adds. "We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease. Here we have demonstrated that the initial steps are feasible."

- here's the release

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