Researchers Discover New Leukemia Genes Through CRISPR Technology

With a survival rate of less than 10% for the most aggressive forms of leukemia, research revolving around the disease has been of paramount importance for scientists. 

Researchers from the University of Rochester have used the latest and most advanced technology known as CRISPR to discover new leukemia genes previously unknown to be part of blood cancers. This data will help paint a clear path to understand how aggressive leukemia arises and grows.

Their findings were published in the journal Nature Cancer.

SEE ALSO: MAGNETIC TOOL THAT REMOVES DISEASES FROM THE BLOOD SET FOR HUMAN TRIALS

Significant discoveries

The research paper pointed to several important discoveries, as per what the University of Rochester scientists pointed out:

• It unveiled a new gene, Staufen 2 (Stau2), that regulates and drives the molecular programs for leukemia stem cells, the cells responsible for propagating the disease and for therapy resistance. Stau2 has been previously studied in the brain and nervous system but until now was not known to have a role in cancer.
• The team used a tool known as CRISPR, which allows scientists to edit DNA in cells and focus on large groups of genes active in a particular disease - in this case, myeloid leukemias. The paper showed that CRISPR can identify an entire class of gene mediators for leukemia, which will aid future research.
• The team also tested its hypothesis in a mouse model designed to mimic the human experience with leukemia, as opposed to conducting studies solely in cell cultures, as several other groups had previously done.

The investigators of this study focused on chronic myeloid leukemia (CML), which can be controlled with specific therapies. However, CML becomes lethal when it advances, or when it is diagnosed in what's known as a "blast" phase.

"This work will be particularly important for the discovery of new treatments," Jeevisha Bajaj, assistant professor of Biomedical Genetics at the University of Rochester, said. "Our genome-wide screen identified cellular signals critical for the growth of cancer, and in the future, this study will be useful to study the microenvironment, the area around the tumor that includes tissue, blood vessels and important molecular signals related to how the cancer behaves."

Before the team discovered Stau2, they found a larger group of RNA-binding proteins (RBPs) and focused on a subset that had not previously been studied in cancer. In doing so, they found that these RBPs were abundant in human leukemia stem cells. Looking further into the matter the team discovered that Stau2 emerged as an important regulator of the growth of CML during the "blast" phase. 

In the lab, the team was able to delete Stau2 and showed that they could block the growth of cancer cells. The next step of the team's research is to see if Stau2 can be targeted for the benefit of the patient.