CHOP and Penn Medicine Launch CRISPR Tool to Study Acute Myeloid Leukemia

New platform allows researchers to test potential cancer targets directly in patient cells

Published on Feb. 27, 2026

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Researchers from Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania's Perelman School of Medicine have developed a CRISPR-based tool that can be used directly on patients' acute myeloid leukemia (AML) cells to identify genes and regulatory elements driving the aggressive blood cancer. This first-of-its-kind approach reveals how individual patient cells respond to genetic changes, making it easier to identify drug targets and understand why some cancers stop responding to treatment.

Why it matters

AML is an aggressive blood cancer that affects the bone marrow and blood, accounting for about one in three leukemias in adults and the second most common blood cancer in children in the U.S. While chemotherapy puts many patients into remission, treatment can be challenging for those whose cancer does not respond or experiences relapse, often due to certain gene or chromosome changes in the leukemia cells. This new CRISPR platform aims to address that challenge by providing more details on the genetic drivers of AML in individual patients.

The details

The researchers developed an efficient way to deliver CRISPR tools directly into primary AML cells taken from patients, achieving high gene-editing efficiency. Using this platform, they screened hundreds of gene edits simultaneously to find edits that reduced or increased cell growth, indicating genes that affect cancer survival. They tested the edits both in vitro and in preclinical models using transplanted patient-derived leukemia cells to validate the findings. The team also combined CRISPR edits with single-cell RNA sequencing to capture the heterogeneous responses of different cells within the leukemia.

  • The findings were published on February 26, 2026 in the journal Molecular Cell.

The players

Junwei Shi, PhD

The study's lead author and an associate professor of Cancer Biology at the University of Pennsylvania's Perelman School of Medicine.

Kai Tan, PhD

A senior study author and a professor in the Department of Pediatrics at Children's Hospital of Philadelphia.

Kathrin M. Bernt, MD

A senior study author and a pediatric oncologist in the Cancer Center's Leukemia and Lymphoma Program at Children's Hospital of Philadelphia.

Children's Hospital of Philadelphia (CHOP)

A non-profit, charitable organization and the nation's first pediatric hospital, founded in 1855.

University of Pennsylvania's Perelman School of Medicine

The nation's first medical school, founded in 1765, and part of Penn Medicine, one of the world's leading academic medical centers.

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What they’re saying

“This platform empowers scientists to test which genes and genetic elements really matter in human tumors. It helps identify drug-ready targets, shows how different tumor subpopulations within the same patient respond and speeds discovery of precision therapies.”

— Junwei Shi, PhD, Lead author and associate professor of Cancer Biology at Penn Medicine (Molecular Cell)

“We have learned that most leukemias are heterogeneous and may contain small subgroups of cells that may ultimately drive poor outcomes. This clarified the results and revealed surprises. We validated previously reported genes that affect leukemia growth but also found that some edits caused cells to die while others halted growth and induced a dormant, therapy‑resistant state. These insights will help prioritize the best candidate genes for therapy development.”

— Kai Tan, PhD, Senior study author and professor in the Department of Pediatrics at CHOP (Molecular Cell)

“Our hope is that this novel platform will identify new ways of developing precision therapies for patients who do not currently have promising options.”

— Kathrin M. Bernt, MD, Senior study author and pediatric oncologist at CHOP (Molecular Cell)

What’s next

The research team plans to tackle other hard-to-treat leukemias next, including pediatric AML.

The takeaway

This new CRISPR-based platform provides a powerful tool for researchers to directly test potential cancer targets in patient-derived leukemia cells, leading to a better understanding of the genetic drivers of AML and informing the development of more effective precision therapies for this aggressive blood cancer.