All-in-one AAV-delivered epigenome-editing platform: proof-of-concept and therapeutic implications for neurodegenerative disorders
B. Kantor, B. Odonovan, J. Rittiner, D. Hodgson, N. Lindner, S. Guerrero, W. Dong, A. Zhang, O. Chiba-Falek
This paper develops a compact AAV-delivered dCas9-based repressor system for gene silencing, demonstrating its effectiveness in reducing ApoE expression in vitro and in vivo.
bioRxiv · Apr 14, 2023 · PreprintBioengineering