Researchers at Penn Medicine and the Children’s Hospital of Philadelphia have developed a new CRISPR tool that can identify the genetic drivers of acute myeloid leukemia (AML) by editing patient cells directly. Unlike traditional lab-grown cell studies, this approach uses real patient samples, capturing the complexity and diversity of actual cancers.
The tool efficiently edits leukemia cells, achieving 86% success for single-gene changes and 73% for multi-gene screening. By testing hundreds of genes at once, researchers can pinpoint which genes fuel cancer growth or cause drug resistance in individual patients. Combining CRISPR with RNA sequencing also allowed them to see how different cell subpopulations respond, revealing that some genetic changes push cells into a dormant state, helping cancers survive chemotherapy and relapse later.
The team plans to expand this platform to other difficult blood cancers, including pediatric AML. Their ultimate goal is to bring the tool into clinical practice, enabling doctors to design personalized treatments that target the unique genetic weaknesses of each patient’s cancer.