New research published in the journal Cell Reports highlights a possible breakthrough in Leukaemia research. Scientists have managed to engineer stem cells to help them understand the role that a specific gene plays in the formation of leukaemia.

Previous research has demonstrated that DNA changes in the gene PTPN11 can cause a disease called Noonan syndrome. The disease has a range of symptoms including unusual facial characteristics, bleeding problems, short stature, heart defects, skeletal malformations and an increase in the risk of getting juvenile myelomonocytic leukaemia (JMML).

Researchers realised they had to understand more about the gene defect to pinpoint what caused the leukaemia. The only current treatment for JMML is a hematopoietic stem cell transplant from bone marrow or umbilical cord blood. The transplant can renew the patient’s ability to generate healthy blood cells. Unfortunately, stem cell transplants for this condition are only effective about half of the time.

Senior author Bruce D. Gelb explains the purpose of the study, saying ”By studying an inherited human cancer syndrome, our study clarified early events in the development of one kind of leukaemia,”

The scientists decided to engineer stem cells from patients with the genetic defect for use in laboratory tests. Researchers took some skin called from patients, then used enzymes to coax the cells into become induced pluripotent stem cells (iPSCs). From there they sent chemical signals to the iPSCs which told them to become hematopoietic stem cells. HSCs are responsible for the creation of blood cells and immune system cells in the human body. Because the original cells came from a patient with the genetic condition, the HSCs also carried the condition.

Scientists could then closely examine how the hematopoietic stem cells worked together to look for a cause for the leukaemia that was common in patients with Noonan syndrome. Isolating stem cells with the PTPN11 gene will help scientists determine the cause of leukaemia.

Source: Scientists engineer stem cells to better understand mechanisms behind leukemia

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