New CRISPR tool disrupts gene in leukemia cells

Myeloblasts with Auer rods seen in acute myeloid leukemia

CRISPR gene editing is a cutting-edge tool that is gaining more and more ground as a way to treat diseases. And as shown in a new study, researchers at the Wellcome Trust Sanger Institute have identified genes that could serve as targets to treat acute myeloid leukemia (AML).

Leukemia cells spread in bone marrow and can be very difficult to control. To create a new method for knocking the cells back, the team of scientists designed a CRISPR/Cas9 technique that would take out all of the genes in the cancer cell individually, eventually narrowing down the ones that would have the greatest effect.

One gene in particular, called KAT2A, when inhibited, disrupted the growth and survival of the leukemia cells without harming surrounding healthy ones. In tests on mice with human leukemia cells, the animals lived longer when KAT2A was disrupted.


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"This is an exciting finding, as KAT2A inhibition worked on a number of primary AML cells with diverse genotypes,” study co-author Konstantinos Tzelepis said in a release. “Whilst the gene needs to be studied in greater depth to understand its potential for use in the clinic, we show that targeting KAT2A destroyed AML cells in the laboratory while sparing healthy blood cells."

The research was funded in part by the Kay Kendall Leukaemia Fund. The results were published in Cell Reports.

"Previous studies showed proof of principle, but this is one of the first systematic attempts to identify the genetic vulnerabilities of AML,” said co-author Kosuke Yusa of the Sanger Institute. “We have improved and applied CRISPR-Cas 9 technology to look at what actually kills cells. CRISPR is becoming a powerful technique in cancer research because it overcomes some of the limitations of earlier tools."

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