Gene Found to Enable Deadly Myelodysplastic Syndromes
Investigators in Cincinnati have linked a gene with myelodysplastic syndromes, potentially offering the first step toward a new genetic therapy.
Myelodysplastic syndromes (MDS) are a group of blood cancers with no known cause in most patients, but in a new study, a group of investigators from the Cincinnati Children’s Hospital Medical Center reports getting closer to solving the mystery.
According to the National Cancer Institute, a division of the National Institutes of Health (NIC),
In most cases of MDS, the
HIF1A essentially tells other genes what to do and plays a key role in how cells respond to metabolic changes and oxygen, affecting more than 1,000 genes; it regulates biological functions in hematopoietic stem cells in the bone marrow, which make blood cells. Using cells donated from MDS patients, the investigators conducted laboratory experiments analyzing the cells and found that dysregulation of HIF1A plays a large role in the onset of MDS, which included a range of patients’ manifestations and symptoms. Further experiments in genetic mouse models assessing the genetic and molecular drivers of MDS confirmed their observations, and inhibiting HIF1A reversed a broad spectrum of MDS symptoms.
“We know the genomes of MDS patients have recurrent mutations in different transcriptional, epigenetic, and metabolic regulators, but the incidence of these mutations does not directly correspond to the disease when it occurs,” said lead author Gang Huang, PhD, in a recent
While extensive additional research will be needed, the study’s authors say their findings indicate that HIF1A is a potential therapeutic target for MDS. Current
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