RNA gene editing in the eye and beyond: The neglected tool of the gene editing armatorium?

Available online 17 August 2022

International Review of Cell and Molecular Biology

Abstract

RNA editing allows correction of pathological point mutations without permanently altering genomic DNA. Theoretically targetable to any RNA type and site, its flexibility and reversibility makes it a potentially powerful gene editing tool. RNA editing offers a host of potential advantages in specific niches when compared to currently available alternative gene manipulation techniques. Unlike DNA editors, which are currently too large to be delivered in vivo using a viral vector, smaller RNA editors fit easily within the capabilities of an adeno-associated virus (AAV). Unlike gene augmentation, which is limited by gene size and viral packaging constraints, RNA editing may correct transcripts too long to fit within a viral vector. In this article we examine the development of RNA editing and discuss potential applications and pitfalls. We argue that, although in its infancy, an RNA editing approach can offer unique advantages for selected retinal diseases.

Keywords

RNA editing

CRISPR

ADAR

CIRTS

LEAPER

BoxB

GluR2

SNAP

Cas13

Gene editing

Gene therapy