<i>CRB1</i>-associated retinal dystrophies in a Belgian cohort: genetic characteristics and long-term clinical follow-up

Abstract

Bi-allelic <i>CRB1</i> mutations result in a range of progressive retinal disorders, most of which are generalised, with characteristically early macular involvement. Visual function and retinal structure analysis indicates a window for potential intervention with gene therapy before the fourth decade of life in RP and the first decade in LCA.