Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
在小鼠和猪动物模型中对视网膜下递送 Cas9 核糖核蛋白的评估突出了基因药物治疗转化的关键考虑因素
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doi:10.1101/2024.12.30.630799
Spencer C Wei, Aaron J Cantor, Jack Walleshauser, Rina Mepani, Kory Melton, Ashil Bans, Prachi Khekare, Suhani Gupta, Jonathan Wang, Craig Soares, Radwan Kiwan, Jieun Lee, Shannon McCawley, Vihasi Jani, Weng In Leong, Pawan K Shahi, Jean Chan, Pierre Boivin, Peter Otoupal, Bikash R Pattnaik, David M