Högskolan i Skövde

Inherited retinal diseases are considered as a leading cause of vision loss in a young population. Neither a permanent cure nor long-term treatment has yet been discovered. However, treating congenital visual impairment by utilizing a sequence-specific nuclease gene editing tool, CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats-associated protein 9), has appeared to have a positive impact in restoring eyesight. The following systematic review was implemented to gain more knowledge about the safety and efficiency features of CRISPR/Cas9 technology when used in clinical trials to treat inherited retinal diseases (IRDs). The review focuses on trials with Leber Congenital Amaurosis and Autosomal Dominant Retinitis Pigmentosa, common childhood IRDs. The studies were synthesized in a proportional meta-analysis using MedCalc software, and supplemented with a narrative literature review, considering both qualitative and quantitative data. The review covers different aspects related tothe use of CRISPR/Cas9s and provides an overview of IRDs and future treatment methods. In conclusion, CRISPR/Cas9, indeed, is seen as a potential technique to treat IRDs. However, different complications do arise, and researchers need to be reminded of the side effects and downsides of CRISPR/Cas9. So, they can further enhance this innovative gene-editing technique in exchange for ultimately achieving long-term treatments for blindness and other inherited diseases.