A new study shows success in using a gene-editing procedure to restore retinal function in mice afflicted by retinitis pigmentosa, according to a recent study in Ophthalmology.¹ The study used electroretinographic and histologic analysis to measure the impact of what the researchers are calling “genome surgery.”^1,2

The technique, known as “clustered regularly interspaced short palindromic repeats” (CRISPR) aims to remove the disease-associated genetic mutation and plant the seeds of a preferred genetic code; in this case, involved the rhodopsin gene. This process—known as “ablate-and-replace”—used “adeno-associated viruses that 1) destroy expression of the endogenous Rho gene in a mutation-independent manner via an improved clustered regularly interspaced short palindromic repeats-based gene deletion and 2) enable expression of wild-type protein via exogenous cDNA.”²

The procedure resulted in both structural and functional improvements.