Retinitis Pigmentosa: Restoring Visual Function With CRISPR/Cas9

Mutated optic rod photoreceptors have been reprogrammed, using the gene-editing tool CRISPR/Cas9, reversing cellular degeneration and restoring visual function in two mouse models of retinitis pigmentosa. The study was done by researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, with colleagues in China.

Retinitis pigmentosa (RP) is a type of inherited vision disorder caused by numerous mutations in more than 60 genes.

The gene mutations affect the eyes’ photoreceptors, specialized cells in the retina that sense and convert light images into electrical signals sent to the brain. There are two types: rod cells that function for night vision and peripheral vision, and cone cells that provide central vision (visual acuity) and discern color. The human retina typically contains 120 million rod cells and 6 million cone cells.

Reversing Retinitis Pigmentosa

In RP, which affects approximately

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