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Ryu, J.* ; Ryder, K.* ; Naka, H.* ; Tran, J.* ; Hinrichs, B.J.* ; Biswas, S.* ; Rust, L.N.* ; Yusova, S.* ; Dozier, B.L.* ; Peterson, S.M.* ; Wettengel, J.M. ; Hanna, C.B.* ; Hennebold, J.D.* ; Burwitz, B.J.* ; Bimber, B.N.*

Generation of a rhesus macaque harboring a multivalent reporter for assessing gene editing outcomes.

Sci. Rep., DOI: 10.1038/s41598-026-52214-2 (2026)
Postprint Forschungsdaten DOI PMC
Open Access Gold möglich sobald Verlagsversion bei der ZB eingereicht worden ist.
Somatic gene editing therapies offer the potential for lasting treatments for a range of genetic disorders, with a host of therapies at various stages of clinical testing. The development of safe, effective therapies required relevant pre-clinical animal models capable of measuring on-target delivery and detecting off-target editing. Here we present the creation of a transgenic rhesus macaque encoding the Bivalent Enhanced Traffic Light Editing (BETLE) system, a multi-color fluorescent reporter that provides a simple readout for multiple modes of gene editing, including off-target effects. We used zygote microinjection and piggyBac transposase to generate preimplantation embryos that ultimately led to a healthy transgenic macaque. Extensive molecular analyses demonstrated single-copy integration of the intact reporter; however, the animal was mosaic, with only 5-8% of cells encoding the reporter, although all germ layers contained positive cells. The creation of this macaque provides important information to improve future transgenics, and progeny from this macaque could provide a powerful tool to evaluate the delivery and accuracy of gene therapies.
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Publikationstyp Artikel: Journalartikel
Dokumenttyp Wissenschaftlicher Artikel
Schlagwörter Rhesus Macaque ; Macaque ; Transgene ; Genome Editing ; Reporter Gene ; Gene ; Gene Targeting ; Zygote ; Electroporation
ISSN (print) / ISBN 2045-2322
e-ISSN 2045-2322
Zeitschrift Scientific Reports
Verlag Springer
Verlagsort London
Begutachtungsstatus Peer reviewed
Institut(e) Institute of Virology (VIRO)
Förderungen NIH Office of the Director