A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina
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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 ther..., , # Data from: A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina
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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 com...,
创建时间：
2026-02-04
