Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo
Yuanbo Qin, Talha Akbulut, Rajakumar Mandraju, Keith Connolly, John Bechill, Farzaneh Assadian, Claudia Foster, Alison Shottek, Seth Levy, Jamie D. Benoit, Tetsuya Yamagata
This paper demonstrates efficient and durable gene activation using Cas9-mediated epigenome editing in a mouse model of LAMA2-CMD, showing sustained therapeutic effects beyond one year.
Human Gene Therapy · Dec 24, 2025Peer-reviewedBioengineering