Allogeneic bone marrow transplant in the absence of cytoreductive conditioning rescues mice with β-thalassemia major

Author:

Huo Yongliang12,Lockhart Jonathan R.1ORCID,Liu Shanrun1,Fontenard Suean1,Berlett Mike1,Ryan Thomas M.1ORCID

Affiliation:

1. Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham, Birmingham, AL; and

2. Key Laboratory of Protein Modification and Degradation, School of Basic Medical Sciences, Affiliated Cancer Hospital & Institute of Guangzhou Medical University, Guangzhou, China

Abstract

Key Points After completion of the fetal-to-adult globin gene switch, humanized β-thalassemia major mice are transfusion dependent. Perinatal humanized β-thalassemia major mice are rescued by bone marrow transplantation in the absence of cytoreductive conditioning.

Publisher

American Society of Hematology

Subject

Hematology

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3. Thalassemia: molecular pathology and management;Steinberg;Am J Med Sci,1988

4. Accelerated programmed cell death (apoptosis) in erythroid precursors of patients with severe beta-thalassemia (Cooley’s anemia);Yuan;Blood,1993

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