BioengineeringHuman Gene TherapyPeer-reviewed
Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo
Yuanbo Qin, Talha Akbulut, Rajakumar Mandraju…·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.
3 citations