Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina
C. Botto, J. Pulman, H. Malki, D. Ren, P. Oudin, A. De Cian, M. As, C. Izabelle, B. Saubamea, S. Fouquet, C. Robert, A. El-Amraoui, S. FISSON, J.-P. Concordet, D. Dalkara
This study delivers Cas9 or base editor RNP complexes directly to retinal cells, achieving gene editing in photoreceptors and RPE, but with toxicity at high concentrations.
bioRxiv · Oct 16, 2023 · PreprintBioengineering