Scaffold-mediated CRISPR-Cas9 delivery system for acute myeloid leukemia therapy

Authors: Tzu‐Chieh Ho, Hye Sung Kim, Yumei Chen, Yamin Li, Mark W. LaMere, Caroline Chen, Hui Wang, Jing Gong, Cal Palumbo, John M. Ashton, Hae‐Won Kim, Qiaobing Xu, Michael W. Becker, Kam W. Leong - Science Advances 2021 cited by 120

knockout reduced LSC colony-forming capacity and leukemic burden. Scaffold-based delivery increased the retention time of LNP-Cas9 in the bone marrow cavity. Overall, sustained local delivery of Cas9/IL1RAP sgRNA via CXCL12α-loaded LNP/MSCM-NF scaffolds provides an effective strategy for attenuating LSC growth to improve AML therapy.