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Blindness-causing genetic defect corrected using CRISPR

More research is necessary to ensure the edits do not introduce other genetic changes before altered stem cells can be transplanted back into a patient.
By Stephen Feller   |   Jan. 27, 2016 at 2:51 PM

NEW YORK, Jan. 27 (UPI) -- Researchers used CRISPR to correct a blindness-causing genetic defect, the first time a defective gene has been successfully fixed in patient-derived stem cells, which may be the first step toward using gene editing to cure a disease.

The successful correction of a gene causing retinitis pigmentosa by researchers at Columbia University is thought to be the first such use of CRISPR, and a significant step on the way to using gene therapy for personalized treatment of health conditions.

Vitamin A is the treatment most commonly used for retinitis pigmentosa, an inherited condition that causes the retina to degrade and leads to blindness. While the treatment slows down the loss of vision, it does not cure the disease.

"Our vision is to develop a personalized approach to treating eye disease," said Dr. Stephen Tsang, an associate professor at Columbia University, in a press release. "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."

For the study, published in Nature: Scientific Reports, researchers took a sample of skin from a patient with retinitis pigmentosa, using it to create stem cells with the patient's DNA in them.

Using CRISPR, the researchers were able to successfully edit the gene RGPR, which is responsible for the degenerative condition. The gene is especially difficult to edit because it contains many repeats and tight-binding nucleotide pairs. Potentially, the cells can be transplanted back into the patient to treat the condition.

While researchers know the cells won't be rejected by the body when transplanted, they said further research is necessary to show the gene editing technique does not introduce unintended modifications to the cells.

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