Gene-editing tool may prevent blindness
A team of researchers is working to turn thepowerful CRISPR gene-editing tool towards treating a serious eyedisease. Early results are promising, with the team successfullycorrecting the mutation that causes the condition in cells outsidethe body.
Retinitis pigmentosa is relativelycommon, affecting around 1 in 4,000 people in the United States andEurope. An inherited condition, it causes the retina to degrade overtime, eventually leading to blindness. The current recommendedtreatment for the condition is for the patient to up their vitamin Aintake, but the measure doesn't cure the disease, instead onlyslowing its progress.
The new research, being conducted byscientists at the Columbia University Medical Center (CUMC) and theUniversity of Iowa, is looking at tackling the condition using a gene-editing tool called CRISPR. The tool has proved extremely useful inrecent 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 createdstem cells from skin samples taken from a single patient with theretinitis pigmentosa. Though grown in the lab, the cells still playhost to the defective gene that causes the condition, which theresearchers were able to correct, outside the body, using the CRISPRtool. The particular type of retinitis pigmentosa used in theexperiment is an excellent candidate for the treatment, as a commonmutation is responsible for 90 percent of cases, making it easier totarget the offending gene.
While the breakthrough may initiallynot seem like a big deal, the treatment potential could be huge. Withthe mutation successfully eradicated from the stem cell, it can thenpotentially be transformed into retinal cells. These could then betransplanted 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 thatthe scientific community has looked to CRISPR to tackle retinitispigmentosa. Earlier this month, a team from the Cedars-Sinai MedicalCenter used the tool to attack the disease in laboratory mice, with very positive results.
The findings of the new research werepublished in the journal Scientific Reports. For more on the study, you can take a look at the video below.