Loss of transgene expression limits liver gene therapy in primates
Jenny A. Greig, Kelly M. Martins, Camilo Ayala Breton, Jason Lamontagne, Yanqing Zhu, Zhenning He, John White, Jingxu Zhu, Jessica A. Chichester, Qi Zheng, Zhe Zhang, Peter L. Bell, Lili Wang, James M. Wilson
This study shows that transgene expression in primate liver declines rapidly after initial transduction, even though vector DNA remains in the nucleus, suggesting that genomic integration may be needed for durable expression.
Nature Biotechnology · Nov 6, 2023Peer-reviewedMolecular Biology