Society for Pediatric Research 2015 Young Investigator Award: genetics of human hematopoiesis-what patients can teach us about blood cell production

Pediatr Res. 2016 Mar;79(3):366-70. doi: 10.1038/pr.2015.245. Epub 2015 Nov 17.

Abstract

Blood cell production or hematopoiesis is one of the most well-understood paradigms of cell differentiation in the body. The majority of work on hematopoiesis comes from studies that have primarily been conducted in mice, zebrafish, or other valuable model systems. However, it is clear that such model organisms may not consistently and faithfully mimic what is observed in humans with blood disorders. Moreover, there is significant divergence between species that is increasingly being appreciated at the genomic level. As a result, there is an opportunity to use observations in humans to provide a refined view of hematopoiesis. Here, we discuss vignettes from our work that illustrate how insight from human genetics can improve our understanding of blood cell production and identify promising therapeutic approaches for blood disorders.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Blood Cells / cytology*
  • Cell Differentiation
  • Erythrocytes / cytology
  • Erythropoiesis
  • Fetal Hemoglobin / metabolism
  • Gene Expression Regulation, Developmental
  • Hematopoiesis / physiology*
  • Hemoglobins / genetics
  • Humans
  • Mice

Substances

  • Hemoglobins
  • Fetal Hemoglobin