BLOG > Publications & Citations > CRISPR-based functional genomics in primary acute myeloid leukemia samples

Authors: Cao Z, Yu S, Peng J et al.
Source: Molecular Cell, 2026; 86, 968-985.e7
We're delighted to share insights from a recent study entitled "CRISPR-based functional genomics for dissecting therapeutic dependency in primary acute myeloid leukemia samples" published in Molecular Cell by Zhendong Cao et al.
Congratulations to all the authors on this excellent article!
They developed an optimized CRISPR-based platform for functional genomics in patient-derived xenograft (PDX) and primary acute myeloid leukemia (AML) samples harboring diverse pathogenic mutations. Integrated in vitro and in vivo CRISPR-Cas9 knockout and CRISPR interference (CRISPRi) dropout screens validated known AML-biased targets and identified cis-regulatory elements essential for leukemic growth. Coupling pooled CRISPR perturbations with single-cell RNA sequencing (Perturb-seq) further resolved the perturbation-induced alterations in regulatory networks, cell cycle states, and cellular hierarchies in primary AML samples.
LentiBlast Premium was used to enhance lentiviral transduction efficiency of PDX and primary Acute myeloid Leukemia (AML) cells, supporting the delivery of large viral vectors such as those containing Cas9.
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