Therapeutic Gene Editing in Inherited Retinal Disorders

Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research has produced several phase 1-2a trials. Here we provide an overview of the mechanisms and applications of various gene-editing technologies including adeno-associated virus vectors, lentiviruses, CRISPR/Cas9 systems, base and prime editing, antisense oligonucleotides, short-hairpin RNAs, Cas13, and adenosine deaminase acting on RNA for the treatment of various inherited retinal diseases (IRDs). We outline the various stages of clinical trials using these technologies and the impacts they have made in advancing the practice of medicine.

Automated summary

Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research has progressed through several phase 1-2a trials. This article provides an overview of the mechanisms and applications of various gene-editing technologies for the treatment of inherited retinal diseases (IRDs), including adeno-associated virus vectors, lentiviruses, CRISPR/Cas9 systems, base and prime editing, antisense oligonucleotides, short-hairpin RNAs, Cas13, and adenosine deaminase acting on RNA. The article also highlights the impact of these technologies on advancing the practice of medicine.