Data from: A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina

CRISPR/Cas9-based gene-editing technologies offer promise for treating
inherited retinal diseases (IRDs); however, safe and efficient ocular
delivery of precision editors remains challenging. To address this
challenge, we report a new class of Coomassie brilliant blue (CBB)-derived
lipidoids that bind and deliver proteins. Subretinal injection of Cre
complexed with these lipidoids into mT/mG mice leads to robust
recombination in the retinal pigment epithelium and photoreceptors. We
employ the CBB-lipidoid platform to deliver adenine base editor (ABE)
ribonucleoproteins (RNP). Incorporating CBB lipidoids into liposomes
improves delivery efficiency. CBB11 stands out for facilitating precise in
vivo ABE-mediated gene editing. Delivery of liposome-CBB11-RNP complexes
results in a 120-fold increase in base editing compared to RNP alone and
restores the scotopic ERG b-wave response in the rd12 mouse model. These
results demonstrate the potential of CBB-augmented, liposome-RNP systems
for therapeutic gene editing in the eye, paving the way for single-dose
precision medicines to treat IRDs.