Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies
突变诱导的神经元和人源化小鼠能够识别尼曼匹克 C1 型蛋白质抑制剂疗法
期刊:JCI Insight
影响因子:6.3
doi:10.1172/jci.insight.179525
Ruth D Azaria, Adele B Correia, Kylie J Schache, Manuela Zapata, Koralege C Pathmasiri, Varshasnata Mohanty, Dharma T Nannapaneni, Brandon L Ashfeld, Paul Helquist, Olaf Wiest, Kenji Ohgane, Qingqing Li, Ross A Fredenburg, Brian Sj Blagg, Stephanie M Cologna, Mark L Schultz, Andrew P Lieberman