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

The hemoglobinopathies are the most prevalent monogenetic disorders and generate substantial medical and socioeconomic burden worldwide [1]. α-Thalassemia major is perinatally lethal and necessitates effective intrauterine intervention to avoid the complications of chronic severe hypoxia evident in transfusion-dependent survivors [2]. β-Thalassemia major and sickle cell disease (SCD) demand substantial resources to prevent permanent or gan failure [3]. Much of the disease burden arises from suboptimal treatment [4].

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