A randomized phase I/II trial of HQK-1001, an oral fetal globin gene inducer, in β-thalassaemia intermedia and HbE/β-thalassaemia

A pilot study of subcutaneous decitabine in  -thalassemia intermedia

(2011) N. F. Olivieri et al.   BLOOD

Novel therapeutic candidates, identified by molecular modeling, induce γ-globin gene expression in vivo

(2011) Michael S. Boosalis et al.   BLOOD CELLS MOLECULES AND DISEASES

Evaluation of Safety and Pharmacokinetics of Sodium 2,2 Dimethylbutyrate, a Novel Short Chain Fatty Acid Derivative, in a Phase 1, Double-Blind, Placebo-Controlled, Single-Dose, and Repeat-Dose Studies in Healthy Volunteers

(2011) Susan P. Perrine et al.   JOURNAL OF CLINICAL PHARMACOLOGY

The inherited diseases of hemoglobin are an emerging global health burden

(2010) D. J. Weatherall   BLOOD

Administration of high doses of recombinant human erythropoietin to patients with β-thalassemia intermedia: a preliminary trial

(2010) K. Bourantas et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study

(2009) A. T. Taher et al.   BLOOD

Discovering the genetics underlying foetal haemoglobin production in adults

(2009) Swee Lay Thein et al.   BRITISH JOURNAL OF HAEMATOLOGY

Erythroid Kruppel-like factor (EKLF) is recruited to theγ-globingene promoter as a co-activator and is required forγ-globingene induction by short-chain fatty acid derivatives

(2009) Susan P. Perrine et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

A genome-wide association identified the common genetic variants influence disease severity in β0-thalassemia/hemoglobin E

(2009) Manit Nuinoon et al.   HUMAN GENETICS

Control of fetal hemoglobin: new insights emerging from genomics and clinical implications

(2009) S. L. Thein et al.   HUMAN MOLECULAR GENETICS

Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of  -thalassemia

(2008) M. Uda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA