Embryonic stem cells as sources of donor-independent platelets
Matthew C. Canver, … , Daniel E. Bauer, Stuart H. Orkin
Matthew C. Canver, … , Daniel E. Bauer, Stuart H. Orkin
Published May 11, 2015
Citation Information: J Clin Invest. 2015;125(6):2261-2263. https://doi.org/10.1172/JCI82348.
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Embryonic stem cells as sources of donor-independent platelets

Abstract

The creation of a donor-independent source of platelets has been challenging; however, recent advances show growing promise for alternative platelet sources. Pluripotent stem cells have the capacity to differentiate into mature megakaryocytes with the ability to produce functional platelets. In this issue of JCI, Noh et al. provide a proof-of-principle demonstration that embryonic stem cells can be used to produce platelets on a clinical scale by controlling the level of the transcription factor GATA1. This study emphasizes the importance of precise regulation of gene expression for regenerative medicine applications.

Authors

Matthew C. Canver, Daniel E. Bauer, Stuart H. Orkin

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Figure 1

Strategy for clinical scale production of platelets ex vivo.

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Strategy for clinical scale production of platelets ex vivo. Engineered ...
Engineered ES cells harbor a doxycycline-inducible promoter that mediates the expression of an shRNA that targets Gata1 mRNA, resulting in degradation of the full-length transcript. Addition of doxycycline induces shRNA-mediated GATA1 suppression, leading to logarithmic proliferation of MEP-like cells (G1ME2 cells). The restoration of GATA1 expression upon doxycycline withdrawal supports differentiation of MEP-like cells into mature megakaryocytes capable of producing functional platelets. TRE, tetracylcine response element.