Gene-editing tool may prevent blindness

Scientists have proved that the gene editing tool CRISPR is effective at combating retinitis pigmentosa in patient-derived stem cells(Credit: Shutterstock)

A team of researchers is working to turn the powerful CRISPR gene-editing tool towards treating a serious eye disease. Early results are promising, with the team successfully correcting the mutation that causes the condition in cells outside the body.

Retinitis pigmentosa is relatively common, affecting around 1 in 4,000 people in the United States and Europe. An inherited condition, it causes the retina to degrade over time, eventually leading to blindness. The current recommended treatment for the condition is for the patient to up their vitamin A intake, but the measure doesn't cure the disease, instead only slowing its progress.

The new research, being conducted by scientists at the Columbia University Medical Center (CUMC) and the University of Iowa, is looking at tackling the condition using a gene-editing tool called CRISPR. The tool has proved extremely useful in recent studies, where it's been tasked with everything from combating muscular dystrophy to helping find new treatments for cancer.

For the new project, the team created stem cells from skin samples taken from a single patient with the retinitis pigmentosa. Though grown in the lab, the cells still play host to the defective gene that causes the condition, which the researchers were able to correct, outside the body, using the CRISPR tool. The particular type of retinitis pigmentosa used in the experiment is an excellent candidate for the treatment, as a common mutation is responsible for 90 percent of cases, making it easier to target the offending gene.

While the breakthrough may initially not seem like a big deal, the treatment potential could be huge. With the mutation successfully eradicated from the stem cell, it can then potentially be transformed into retinal cells. These could then be transplanted back into the patient to treat the vision loss, with little risk of the cells being rejected by the host.

"We still have some way to go, but we believe that the first therapeutic use of CRISPR will be to treat an eye disease," said CUMC associate professor Stephen Tsang. "Here we have demonstrated that the initial steps are feasible."

This study isn't the first time that the scientific community has looked to CRISPR to tackle retinitis pigmentosa. Earlier this month, a team from the Cedars-Sinai Medical Center used the tool to attack the disease in laboratory mice, with very positive results.

The findings of the new research were published in the journal Scientific Reports. For more on the study, you can take a look at the video below.

Source: CUMC

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