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A human H1-HBB11-GFP reporter embryonic stem cell line (WAe001-A-2) generated using TALEN-based genome editing. | LitMetric

A human H1-HBB11-GFP reporter embryonic stem cell line (WAe001-A-2) generated using TALEN-based genome editing.

Stem Cell Res

Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Department of Cellular, Developmental, and Regenerative Biology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Electronic address:

Published: May 2020

AI Article Synopsis

  • Hemoglobin production in mammals involves switching between different globin gene expressions as they develop, impacting red blood cell functionality.
  • Disruptions in this gene switching can result in blood disorders like sickle cell disease and β-thalassemia.
  • The study introduces a human embryonic stem cell line modified to express an adult globin reporter, enabling researchers to test small molecules that could help produce adult-type hemoglobin.

Article Abstract

Hemoglobin production during mammalian development is characterized by temporal switches of the genes coding for the α- and ß-globin chains. Defects in this controlled process can lead to hemoglobinapathies such as sickle cell disease and ß-thalassemia. The ability of human embryonic stem cells (hESC) to proceed through hematopoiesis could provide a clinically useful source of red blood cells. However, hESC-derived red cells exhibit an embryonic/fetal, but not adult, mode of hemoglobin expression. The resource described here is a hESC line engineered to express a reporter from its adult globin promoter, providing a screening platform for small molecules that lead to efficient induction of adult globin.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7297435PMC
http://dx.doi.org/10.1016/j.scr.2020.101837DOI Listing

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