A research team led by Icahn Mount Sinai School of Medicine (Icahn Mount Sinai) established the first cell model to describe the evolution of acute myeloid leukemia (AML) from early to late.
The results of this research were published in the February issue of Cell Stem Cell. Researchers used gene editing technology to change the genes that make cells malignant, so that they can identify potential therapeutic targets in the early stages of the disease.
The therapeutic target can be applied not only to AML, but also to blood cancer myelodysplastic syndrome and clonal hematopoiesis, which is usually a pre-leukemia disease.
"We built a leukemia model from the ground up that characterizes the molecular changes in disease progression, allowing us to identify the earliest events that will develop into therapeutic targets," said Eirini Papapetrou, MD, professor of oncology at Icahn Mount Sinai University Said, “By creating the first cell model to track the evolution of human leukemia, we believe that we have taken an important step towards uncovering the cell biology of the disease. We have identified the molecular vulnerability that occurs early in the disease process, This may lead to improved biomarkers and novel treatments for AML. These targets have confused the medical community in the past.”
AML is a cancer of the blood and bone marrow, which is the spongy tissue inside the bones that make blood cells. The disease mainly affects white blood cells and is one of the most common leukemias in adults, with more than 20,000 newly diagnosed cases in the United States each year. Although several new drugs have been introduced for the disease in recent years, they have not significantly changed the prognosis or survival rate of patients.
The researchers introduced specific gene mutations that cause leukemia through CRISPR-mediated gene editing, and they determined to better understand the successive stages of leukemia evolution. CRISPR is a powerful tool that allows the research team to change the DNA sequence in induced pluripotent stem cells (iPSC) to create a model with an ever-increasing number of mutant genes that appear to correspond to human myeloid diseases The progressive malignant characteristics. CRISPR gene editing uses Cas9, this enzyme acts like a pair of molecular scissors, with the ability to cut DNA strands. By using these tools, researchers can outline the gradual transformation of normal cells into malignant cells.
Dr. Papapetrou explained: "CRISPR / Cas9 and iPSC technology provides us with a unique opportunity to characterize the change in the basis of transitions between the various stages of AML, and use patterns of these changes to determine the target gene for early intervention."
Specifically Said that the researchers found that inflammation and innate immune pathways constitute such early targets, and proved that inhibitors of these pathways, and therapies being tested in blood cancer and immune-related diseases may be promising for AML and myelodysplastic syndromes. Of treatment.
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