Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration by Fry, LE, Major, L, Salman, A, McDermott, LA, Yang, J, King, AJ, McClements, ME, MacLaren, RE

“…We develop and characterize a mouse model of Usher syndrome carrying the c.11840 G > A mutation designed for the evaluation of base editors for inherited retinal disease. …”

Development of CRISPR gene therapy for retinal degenerations by Fry, LE

“…Subsequently, an Ush2a-W3947X mouse model of the syndromic IRD Usher syndrome was developed and characterised. I finally used subretinal, AAV-delivered RNA base editors to successfully correct the molecular phenotype of the homozygous Ush2a-W3947X mouse.…”