Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo
Y. Qin, T. Akbulut, R. Mandraju, K. Connolly, J. Bechill, F. Assadian, A. Shottek, S. Levy, J. Benoit, T. 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.
bioRxiv · May 3, 2024 · PreprintBioengineering