Potential CRISPR base editing therapeutic options in a Sorsby fundus dystrophy patient by Elsayed, MEAA, Kaukonen, M, Kiraly, P, Kapetanovic, JC, MacLaren, RE

In silico analysis of pathogenic CRB1 single nucleotide variants and their amenability to base editing as a potential lead for therapeutic intervention by Bellingrath, J-S, McClements, ME, Kaukonen, M, Fischer, MD, MacLaren, RE