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注射的 Cas9 核糖核蛋白进行评估，突显了基因药物治疗转化中的关键考虑因素
期刊:PLoS One
影响因子:2.6
doi:10.1371/journal.pone.0317387
Wei Spencer C, Cantor Aaron J, Walleshauser Jack, Mepani Rina, Melton Kory, Bans Ashil, Khekare Prachi, Gupta Suhani, Wang Jonathan, Soares Craig, Kiwan Radwan, Lee Jieun, McCawley Shannon, Jani Vihasi, Leong Weng In, Shahi Pawan K, Chan Jean, Boivin Pierre, Otoupal Peter, Pattnaik Bikash R, Gamm David M, Saha Krishanu, Gowen Benjamin G, Haak-Frendscho Mary, Janatpour Mary J, Silverman Adam P
