SNHS Spotlight: CRISPR Gene Editing Used to Treat Leukemia

CRISPR is a scientific technology that is used to edit genes. It works by either altering pieces of DNA or turning genes on or off without altering the sequence. A major component of the CRISPR technology is the many different “Cas” proteins found in bacteria. The most common protein, Cas9, helps defend against viruses and is easily used by giving it a piece of RNA to guide it to any target sequence. CRISPR is utilized in many different scientific fields to edit the genomes of plants and animals and even some medicines. CRISPR can also be used to prevent some diseases or to treat them. One disease that is now being researched for CRISPR to treat is acute myeloid leukemia. 

crispr screening
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Acute myeloid leukemia is cancer found in the blood and the bone marrow. This type of cancer begins in bone marrow, spreads into the blood, and sometimes even moves to other parts of the body if not treated. Myeloid stem cells can make red blood cells, granulocytes (white blood cells), or platelets. In acute myeloid leukemia, these stem cells become an immature white blood cells called myeloblasts, abnormal red blood cells, or platelets, instead of their normal forms. These cells are called leukemia cells which build up in the bone marrow and blood so there is less room for the healthy cells. This can cause infection, anemia, or easy bleeding. 

Scientists have been working to identify a treatment for AML that would leave patients with fewer side effects using CRISPR technologies. These scientists are targeting ZMYND8, a regulatory protein that cancer cells need to control gene expression to keep them alive and growing. Scientists have used CRISPR to discover that inhibiting the epigenetic reader function of ZMYND8 in mice left them with smaller tumors and better survival. The epigenetic reader’s function is to use proteins to identify and interpret modifications within a specialized domain. Researchers continued on to find a biomarker to predict the sensitivity of cancer cells to a ZMYND8 inhibitor. Overall, researchers have made some advancements with the CRISPR technology regarding acute myeloid leukemia, but there is still work to be done to finalize this treatment.

Works Cited 

National Cancer Institute: New Scientist: 

Penn Medicine News: new-drug-target-for-leukemia

By: Madyson Ayers’24, SNHS Member

Categories: SNHS