A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model

•The HbbTh3/+ mouse is a good model of severe thalassemia for in utero therapy.•In utero and postnatal transplantation with immunosuppression resulted in better chimerism.•In utero gene therapy produced partial hematological correction but not full rescue.•Both strategies need further optimization to overcome the hostile microenvironment. Major hemoglobinopathies place tremendous strain on global resources. Intrauterine hemopoietic cell...