New Technology Could Cure Retinitis Pigmentosa (RP)

CRISPR/cas9 gene diagram

By Hillary Kleck

A revolutionary new technology called CRISPR, designed for gene-editing, is being used to research a possible treatment for retinitis pigmentosa (RP) by Columbia University Medical Center and the University of Iowa. A recent study published in Scientific Reports identifies a promising breakthrough using stem cells from skin samples containing the mutated RGPR genes – one of the most common causes of the eye disease RP.

Researchers successfully used the CRISPR technology to repair the defective genes of a rat. This is exciting because the potential lies in the possibility that these repaired genes can now be placed back into the donor eye as a cure for retinitis pigmentosa. It could very well be the first personalized treatment for humans in the future with a lessened fear of the body rejecting the edited cells since they are going back into the original donor. Other eye diseases caused by mutated genes could also be treated using the same method as well.

The CRISPR technology is not yet approved for human use but steps are being taken so that it can possibly be approved one day in the near future. Researchers describe this finding as promising since the gene they successfully edited is relatively complex so other simpler genes could possibly be edited even easier. There’s obviously a lot to be done before anything is certain, but there is hope!

Read the full article on the Columbia University Medical Center’s Newsroom. The referenced study can be found on Scientific Reports. If you’d like to learn more about the CRISPR/Cas9 technology for gene-editing, watch this TED Talk video below of one of the technology’s co-inventors:



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