Multiplex HDR for Disease and Correction Modeling of SCID by CRISPR Genome Editing in Human HSPCs
O. Iancu, D. Allen, O. Knop, Y. Zehavi, D. Breier, A. Arbiv, A. Lev, Y. N. Lee, K. Beider, A. Nagler, R. Somech, A. Hendel
This paper models SCID and tests gene correction in human HSPCs using CRISPR-Cas9 and multiplexed HDR, showing successful T-cell development from corrected cells.
bioRxiv · Oct 7, 2022Bioengineering