Mitchell Weiss, M.D., Ph.D.

Mitchell Weiss, M.D., Ph.D.

Professor of Pediatrics

Department of Pediatrics

The Children's Hospital of Philadelphia
The Abramson Research Center 316B
3615 Civic Center Blvd.
Philadelphia, PA 19104


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We are a molecular hematology lab interested in how transcription factors control blood cell development. We use mutant mice, embryonic stem (ES) cells, induced pluripotent stem cells (iPSCs), and tissue culture cells to perform our studies. We are interested in how DNA binding proteins control the survival, proliferation, and differentiation of hematopoietic stem and progenitor cells. Our studies are relevant to understanding both how normal blood cells form and mechanisms of leukemic transformation. Major projects in our laboratory include:

  • Creating and manipulating induced pluripotent stem cells (iPSCs) and human embryonic stem (ES) cells to create improved models for understanding and manipulating normal and pathological hematopoiesis.
  • The role of microRNAs in hematopoiesis. We are examining miR144/451, a microRNA locus that is expressed abundantly in red blood cell precursors. We showed that knockout of this locus inhibits red blood cell development in mice and zebrafish. We are studying the associated mechanisms by identifying functional miR144/451 target genes and their relevant pathways.
  • The role of transcription factor GATA-1 in Down syndrome (DS) associated leukemogenesis. Acute megakaryoblastic leukemia is associated with trisomy 21 and mutations in the GATA1 gene. We are studying gene-manipulated mice and samples from Down syndrome patients to better understand how these two genetic lesions synergize in malignant transformation.
  • We discovered alpha hemoglobin stabilizing protein (ASHP), a molecular chaperone that regulates hemoglobin production and homeostasis. We are using biochemical and biophysical approaches to understand how AHSP facilitates folding and stabilization of a globin. In related work, we are examining how developing red blood cells control hemoglobin and other proteins through protein quality control mechanisms including proteolysis, molecular chaperones and autophagy.
  • We are performing genome-wide studies to better define how GATA-1 cooperates with cofactor proteins to modify chromatin and regulate gene expression during erythroid and megakaryocytic differentiation. This work complements our studies of Down syndrome associated leukemia, where the GATA1 gene is mutated.


Home Participating Investigators Mitchell Weiss, M.D., Ph.D.