CRISPR: On the road to restoring sight to the blind

Abstract

CRISPR-Cas9 is a novel gene-editing tool that promised to revolutionize our ability to treat genetic conditions when first introduced. Today, it continues to fuel many areas of health research, ranging from cancers to sickle cell disease to Huntington’s disease.1–3 Vision science researchers immediately saw the potential of CRISPR, with some of the earliest experiments exploring CRISPR as a treatment option for inherited ocular disorders.4 Only a few years later, in March 2020, vision research was again at the forefront of this field.5 A CRISPR therapy was injected into the human body for the first time in an attempt to correct a vision threatening mutation.5 Inherited retinal diseases have traditionally presented therapeutic challenges, but CRISPR is now providing hope for a cure.