Sickle Human Umbilical Cord Derived Erythroid Progenitor Cells (S-HUDEP2): An Ideal in-Vitro System for Screening Anti-Sickling Compounds for Sickle Cell Disease

Conclusions: S-HUDEP2 cells express HbS, form DRBC, and sickle under hypoxic conditions, just like erythroid precursors and mature red blood cells from individuals with SCD. Exposure to two anti-sickling agents, one of which is currently in Phase III clinical trials, significantly decreased S-HUDEP2 sickling under hypoxic conditions, and reduced %DRBC, a marker of disease severity. Advantages of S-HUDEPs over patient samples include genetic and phenotypic uniformity, no human pathogens, and availability to groups without access to patient samples. We therefore conclude that S-HUDEP2s have utility in clinical research, may be used to screen anti-sickling and anti-dense cell compounds in vitro, and may lead to identification of new therapeutic options for SCD patients.DisclosuresNo relevant conflicts of interest to declare.

Source: Blood - November 21, 2018 Category: Hematology Authors: Chang, A., Park, S. H., Lee, C. M., Paikari, A., Bao, G., Sheehan, V. A. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster III Source Type: research