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

• HbbTh3/+ mouse is a good model of severe thalassaemia for in-utero therapy• Better chimerism after in-utero and postnatal transplantation with immunosuppression• In-utero gene therapy produced partial haematological correction but not full rescue• Both strategies need further optimisation to overcome hostile microenvironment

Source: Experimental Hematology - March 29, 2018 Category: Hematology Authors: Niraja M. Dighe, Kang Wei Tan, Lay Geok Tan, Steven S.W. Shaw, Suzanne M.K. Buckley, Dedy Sandikin, Nuryanti Johana, Yi-Wan Tan, Arijit Biswas, Mahesh Choolani, Simon N. Waddington, Michael N. Antoniou, Jerry K.Y. Chan, Citra N.Z. Mattar Tags: Brief Communication Source Type: research