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.

 1. Tsai Y, Wu W, Lee T, et al. Clustered regularly interspaced short palindromic repeats-based genome surgery for the treatment of autosomal dominant retinitis pigmentosa. Ophthalmology. 2018. https://www.aaojournal.org/article/S0161-6420(17)33608-4/fulltext. Accessed May 14, 2018.
2. American Academy of Ophthalmology. Genome surgery for eye disease moves closer to reality: Study shows that a CRISPR-based treatment can restore retinal function in mice. ScienceDaily. 12 May 2018. www.sciencedaily.com/releases/2018/05/180511102401.htm. Accessed May 14, 2018.