Open AccessGaucher Disease‐Induced Pluripotent Stem Cells Display Decreased Erythroid Potential and Aberrant Myelopoiesis
Author(s) -
Sgambato Judi A.,
Park Tea Soon,
Miller Diana,
Panicker Leelamma M.,
Sidransky Ellen,
Lun Yu,
Awad Ola,
Bentzen Søren M.,
Zambidis Elias T.,
Feldman Ricardo A.
Publication year - 2015
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.5966/sctm.2014-0213
Subject(s) - myelopoiesis , haematopoiesis , myeloid , induced pluripotent stem cell , biology , cd34 , glucocerebrosidase , stem cell , immunophenotyping , progenitor cell , lysosomal storage disease , immunology , pathology , microbiology and biotechnology , cancer research , embryonic stem cell , medicine , genetics , antigen , enzyme , biochemistry , gene
Induced pluripotent stem cells (iPSCs) derived from patients with types 1, 2, and 3 Gaucher disease (GD) were differentiated to CD34 + /CD45 + /CD43 + /CD143 + hematopoietic progenitor cells and their developmental potential was examined. The results suggest that GD hematopoietic progenitors have decreased erythroid potential as well as increased and aberrant myelopoiesis. Abnormal myelopoiesis might contribute to the pathological properties of Gaucher macrophages, central to GD manifestations. The hematopoietic developmental defects observed reflect hematologic abnormalities in GD patients, demonstrating the utility of GD‐iPSCs for modeling this disease.