PRPF31 #RetinitisPigmentosa is a #retinal disease caused by haploinsufficiency. #PRPF31 mutations are also homozygous lethal, which makes creating an animal model challenging. We tackled this problem by knocking out PRPF31 using a #CRISPR construct in adults using #AAV – and then showed that #GeneTherapy could prevent retinal degeneration in vivo. Out now in #NatureCommunications. https://www.nature.com/articles/s41467-022-35361-8