Researchers from the University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, as well as colleagues in China, have used the gene-editing too CRISPR/Cas9 to reprogram mutated rod photoreceptors to become functioning cone photoreceptors, reversing cellular degeneration and restoring visual function in two mouse models of retinitis pigmentosa (RP).
The research team was reportedly able to use CRISPR/Cas9 to deactivate a master switch gene called Nrl and a downstream transcription factor called Nr2e3, which reprogrammed rod cells to become cone cells. According to researchers, cone cells are less vulnerable to the genetic mutations caused by RP.
Upon testing their approach in two different mouse models of RP, the scientists reportedly found an abundance of reprogrammed cone cells and preserved cellular architecture in the retinas. And electroretinography testing of rod and cone receptors in live mice show improved function.
The study was recently published online in the journal Cell Research.
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Source: University of California San Diego