Efficient in vivo genome editing mediated by stem cells-derived extracellular vesicles carrying designer nucleases
S. Bobis-Wozowicz, K. Kania, K. Nit, N. Blazowska, K. Kmiotek-Wasylewska, M. Paw, E. Karnas, A. Szyposzynska, M. Tyszka-Czochara, O. Woznicka, D. Boruczkowski, C. Mussolino, P. P. Łabaj, A. Schambach, Z. Madeja, , E. Zuba-Surma
This study demonstrates that stem cell-derived extracellular vesicles can deliver designer nucleases in vivo, achieving up to 50% gene knockout in the liver.
bioRxiv · Feb 25, 2021 · PreprintBioengineering