Recent advances in gene editing technology have introduced the potential for application of mutagenesis approaches in nonhuman primates to model human development and disease. Here we report successful TALEN-mediated mutagenesis of an X-linked, Rett syndrome (RTT) gene, methyl-CpG binding protein 2 (MECP2), in both rhesus and cynomolgus monkeys. Microinjection of MECP2-targeting TALEN plasmids into rhesus and cynomolgus zygotes leads to effective gene editing of MECP2 with no detected off-target mutagenesis. Male rhesus (2) and cynomolgous (1) fetuses carrying MECP2 mutations in various tissues including testes were miscarried during midgestation, consistent with RTT-linked male embryonic lethality in humans. One live delivery of a female cynomolgus monkey occurred after 162 days of gestation, with abundant MECP2 mutations in peripheral tissues. We conclude that TALEN-mediated mutagenesis can be an effective tool for genetic modeling of human disease in nonhuman primates. © 2014 Elsevier Inc.
CITATION STYLE
Liu, H., Chen, Y., Niu, Y., Zhang, K., Kang, Y., Ge, W., … Ji, W. (2014). TALEN-mediated gene mutagenesis in rhesus and cynomolgus monkeys. Cell Stem Cell, 14(3), 323–328. https://doi.org/10.1016/j.stem.2014.01.018
Mendeley helps you to discover research relevant for your work.