Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients

3'UTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice

(2011) K. Ikeda et al.   BLOOD

A preclinical approach for gene therapy of β-thalassemia

(2010) Laura Breda et al.   Annals of the New York Academy of Sciences

Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in β-thalassemia

(2010) Michel Sadelain et al.   Annals of the New York Academy of Sciences

Mutation of a barrier insulator in the human ankyrin-1 gene is associated with hereditary spherocytosis

(2010) Patrick G. Gallagher et al.   JOURNAL OF CLINICAL INVESTIGATION

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

The 3′ Region of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to Its Core and Is Required for Full Insulator Activity

(2009) Paritha I. Arumugam et al.   PLoS One

Resolving the distinct stages in erythroid differentiation based on dynamic changes in membrane protein expression during erythropoiesis

(2009) K. Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

ON THE ROAD TO GENE THERAPY FOR β-THALASSEMIA AND SICKLE CELL ANEMIA

(2008) Arthur Bank   PEDIATRIC HEMATOLOGY AND ONCOLOGY

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA