AUG 24, 2021 4:00 PM PDT

CRISPR Helps Identify New Therapeutic Target for Leukemia

WRITTEN BY: Ryan Vingum

Researchers may have found a new druggable target in patients with acute myeloid leukemia (AML), thanks to a CRISPR screening tool. 

According to a study published in Molecular Cell, researchers identified the ZMYND8 gene as a possible therapeutic target for patients with AML. The study’s lead author, Junwei Shu, stated that “cancer cells in patients with AML rely heavily on ZMYND8, and thanks to a sophisticated CRISPR-based screening approach, we pinpointed the exact 'druggable pocket' to target.” Specifically, researchers found that ZMYND8, though not a mutated gene, was a necessary component of helping cancer cells stay alive through regulation of gene expression. 

CRISPR is well known as a tool that has had many uses in the past, though most notably, it has been used to locate specific parts of a DNA sequence and edit or replace them for therapeutic benefit. Researchers in this study used CRISPR to actually map and then disrupt protein function in cancer cells, allowing them to catalogue proteins and modify how these proteins were used. The research team specifically focused on disrupting the epigenetic reader function of the ZMYND8 gene. When tested in mice, researchers were able to produce smaller tumors, suggesting the potential of both the CRISPR tool to screen for future therapeutic targets and the therapeutic potential of ZMYND8 in AML patients. CRISPR also helped identify biomarkers that could help predict if a cancer cell would be responsive to a drug designed to inhibit ZMYND8 activity, which may lead to more specific, effective treatments in the future. 

This use of CRISPR offers a way of developing targeted therapy, or therapy designed for specific enzymatic and genetic targets. While this approach is not new, CRISPR appeared to locate previously unknown targets underlying cancers such as AML. This could allow for wider discovery of druggable targets in cancer cells. And while AML only affects about .5% of adult men and women, the National Cancer Institute notes that the rate of new cases has been rising over the years, highlighting a need for new therapeutic options. CRISPR may be one such tool in the search for new cancer treatments.

Source: Science Daily; National Cancer Institute; European Journal of Pharmacology; Molecular Cell

About the Author
Master's (MA/MS/Other)
Science writer and editor, with a focus on simplifying complex information about health, medicine, technology, and clinical drug development for a general audience.
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