The First Affiliated Hospital and Center for Stem Cell and Regenerative Medicine, Department of Basic Medical Sciences, College of Medicine, Zhejiang University, Hangzhou, Zhejiang, 310058, China; Institute of Hematology, Zhejiang University, Hangzhou, Zhejiang, 310058, China; Translational Systems Biology Group, Department of Comparative Biology and Safety Sciences, Amgen Inc., Cambridge, MA 02141, USA.
Center for Individualized Medicine, Department of Molecular Pharmacology & Experimental Therapeutics, Mayo Clinic, Rochester, MN 55905, USA.
Cell Stem Cell. 2017 Aug 3;21(2):161-165. doi: 10.1016/j.stem.2017.07.012.
Merging iPSC models and human genetic research has opened up new avenues in understanding disease mechanisms and target biology, which facilitate exciting translation of this research to many areas of drug development. We highlight recent applications of these combined disciplines and discuss remaining challenges and potential solutions.
将 iPSC 模型与人类遗传研究相结合,为理解疾病机制和靶标生物学开辟了新途径,这为将该研究转化为药物开发的许多领域提供了令人兴奋的机会。我们重点介绍了这些综合学科的最新应用,并讨论了仍然存在的挑战和潜在的解决方案。