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<p>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.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月19日

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  • Junghyun Ryu,
  • Kristie Ryder,
  • Hiroaki Naka,
  • Jenni Tran,
  • Brayden J. Hinrichs,
  • Sreya Biswas,
  • Lauren N. Rust,
  • Sofiya Yusova,
  • Brandy L. Dozier,
  • Samuel M. Peterson,
  • Jochen M. Wettengel,
  • Carol B. Hanna,
  • Jon D. Hennebold,
  • Benjamin J. Burwitz,
  • Benjamin N. Bimber

摘要

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.