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… blood cancers such as myelodysplastic syndrome or leukemia. Newsletter 2 Genomic research team New additions to … This meant restricting patients to those who are having acute interventions performed such as bone marrow transplant, … with R U N X1 to increase a person’s risk of developing leukemia or make their disease more severe? Are there …
News Release
NHGRI researchers have discovered a new inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS), which is caused by mutations in the UBA1 gene.
… and vacuoles (unusual cavity-like structures) in myeloid cells. The scientists reported their findings in the … that the mosaicism was indeed present in the patients’ myeloid cells, which are responsible for systemic … and vacuoles (unusual cavity-like structures) in the myeloid cells. Out of the combined 25 individuals, …
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David Bodine, founder of the Hematopoiesis Section and chief of the Genetics and Molecular Biology Branch, advanced the fields of gene therapy and hematology in his 38 years at the NIH. Now, he plans to continue his contributions to science as an emeritus and through training the next generation of scientists.
… and ask, “Why does that one stem cell go nuts and become leukemia? Is it because of where it is, or is it because of … If you could then change the microenvironment and cure leukemia that way, it'd be a lot better than chemotherapy. … …
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… ministudy@nih.gov National Human Genome Research Institute Acute Infection in Mitochondrial Disease Why are we doing … Research Nurse and study coordinator, at 301-451-9145. … Acute Infection in Mitochondrial Disease Study Flyer - …
Educational Resources
Gene therapy is an experimental technique for treating disease by altering the patient's genetic material.
… and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy. … Educational …
Staff
Dr. Gordon-Lipkin is a researcher and child neurologist with expertise in neurodevelopmental disabilities in the Metabolism, Infection and Immunity (MINI) Section.
… conditions including autoimmune encephalitis, acute flaccid myelitis, and multiple sclerosis. Her current … neuroimaging, and functional profiles in children with acute flaccid myelitis at acute and convalescent stages of disease .  Dev Med Child …
News Release
The zebrafish is a powerhouse tool that helps researchers better understand the genes that are implicated in disease.
… changes that lead to certain blood cancers, such as leukemia. Although Dr. Liu acknowledged that the zebrafish is not a proven model for leukemia, its transparent embryos develop in the water away …
Archive
… Researchers discover key mutation in acute myeloid leukemia … Archive …
Staff
Dr. Amanda Ombrello is a clinical researcher in the Inflammatory Disease Section of NHGRI's Medical Genetics Branch.
… AK . Cryopyrin-associated periodic syndrome caused by a myeloid-restricted somatic NLRP3 mutation . Arthritis …
Staff
Dr. Hanchard is a clinical investigator within the Center for Precision Health Research (CPHR) and head of the Childhood Complex Disease Genomics Section within the CPHR.
… medicine by going from bedside to bench and back. … Severe Acute Malnutrition (SAM) Approximately two million children … 20-30% of whom do not survive. Classically, severe acute malnutrition is described as being either non-edematous … M Manary, I Trehan, NA Hanchard^. Edematous severe acute malnutrition is characterized by hypomethylation of …