Licensing status

Publication and contact information

Disease models

Transcription activator-like effector nuclease (TALEN)-mediated genome editing to generate genetically modified monkeys

Primate studies suggest TALENs could be used to generate genetically modified monkeys to model disease. In single-cell rhesus or cynomolgus monkey embryos, microinjection of three methyl CpG binding protein 2 (MECP2; RTT)-targeting TALEN plasmids plus a RAD51 homolog (RAD51) plasmid resulted in eight-cell-stage embryos with MECP2 disruptions. Embryo transfer to female monkeys produced three aborted male fetuses and one live female infant. Sequencing analysis of tissues from the TALEN monkeys showed that the TALEN fetuses and infant had multiple point mutations across MECP2 and no off-target mutational effects. Next steps include using the system to generate monkey models for neurological and metabolic diseases.

SciBX 7(10); doi:10.1038/scibx.2014.296
Published online March 13, 2014

Unpatented; licensing status not applicable

Liu, H. et al. Cell Stem Cell; published online Feb. 13, 2014;
Contact: Weizhi Ji, Yunnan Key Laboratory of Primate Biomedical Research, Kunming, China
Contact: Yi Eve Sun, same affiliation as above
Contact: Siguang Li, Tongji University School of Medicine, Shanghai, China