High-level embryonic globin production with efficient erythroid differentiation from a K562 erythroleukemia cell line

A widely available cell line with erythroid differentiation capability is a useful tool with which to analyze red blood cell (RBC) biology and develop new genetic strategies for RBC diseases. Genetic modification of erythroid progenitor cells and hematopoietic stem cells (HSCs) is potentially curative for hemoglobin disorders, including β-thalassemia and sickle cell disease (SCD). In current gene therapy trials for hemoglobin disorders, a correct β-globin gene is transferred to HSCs and the gene-modified HSCs are transplanted to patients [1,2].

Source: Experimental Hematology - March 7, 2018 Category: Hematology Authors: Naoya Uchida, Juan J. Haro-Mora, Selami Demirci, Atsushi Fujita, Lydia Raines, Matthew M. Hsieh, John F. Tisdale Source Type: research