First CRISPR-based platform to pinpoint leukemia drivers
The new toolkit, developed by Penn Medicine and Children’s Hospital of Philadelphia, allows researchers to test potential cancer targets directly in patient leukemia cells.
2 min. read
A new CRISPR-based tool that is directly used on patients’ cancer cells can identify genes and regulatory elements driving acute myeloid leukemia (AML), an aggressive blood cancer affecting the bone marrow and blood. This first-of-its-kind approach reveals how individual patient cells respond to genetic changes and makes it easier to identify drug targets and understand why some cancers stop responding to treatment. The findings are published in Molecular Cell, by researchers from Penn Medicine and Children’s Hospital of Philadelphia (CHOP).
CRISPR genome-editing tools allow researchers to quickly test hundreds of genes at the same time to determine which ones are important for cancer growth and survival. These genetic toolkits have enabled the discovery and validation of new cancer drug targets. Until now, this approach has mostly been used in preclinical models or in long-established cancer cell lines grown in labs. These models do not fully represent the genetic diversity seen in patients. Researchers sought to apply CRISPR tools on heterogeneous tumor samples from patients to better understand which genetic elements cancer cells depend on and how they would respond to treatment.
Because the new tool works on patient samples, the researchers hope it could one day be used not just as a research tool, but in the clinical setting to better prioritize treatment options based on each patient’s cancer’s unique biology.
In this study, the team developed a more efficient way to deliver CRISPR tools into AML cells taken from patients. They optimized viral vectors and delivery methods to introduce CRISPR components directly into primary leukemia cells, achieving high gene-editing efficiency. Using this platform, the team screened hundreds of gene edits simultaneously to find edits that reduced or increased cell growth, indicating genes that affect cancer survival. They tested edits both in vitro and in preclinical models using transplanted patient-derived leukemia cells to validate the findings.
“This platform empowers scientists to test which genes and genetic elements really matter in human tumors,” says Junwei Shi, the study’s lead author, and an associate professor of cancer biology in the Perelman School of Medicine. “It helps identify drug-ready targets, shows how different tumor subpopulations within the same patient respond and speeds discovery of precision therapies.”
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