Hematology

Mouse studies suggest an shRNA-microRNA construct targeting BCL11A in erythroid cells could help treat SCD. The construct consisted of a BCL11A-targeting shRNA sequence incorporated into a microRNA (miRNA) backbone under the control of a Pol II promoter for erythrocyte-specific expression. In lethally irradiated mice receiving hematopoietic stem cells (HSCs) from a mouse model of SCD, pretreatment of the HSCs with a lentiviral vector encoding the BCL11A-targeting construct increased erythrocyte counts, hematocrit and levels of plasma hemoglobin and decreased reticulocyte counts and spleen weight compared with lentiviral vectors encoding inactive fluorescent proteins. Next steps could include testing the construct in additional models of SCD...