Future alternative therapies for β-thalassemia

Control of homozygous β-thalassemia by carrier screening and antenatal diagnosis in Sardinians

(2010) A. Cao et al.   CLINICAL GENETICS

Production of β-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous β039 thalassemia patients

(2009) Francesca Salvatori et al.   AMERICAN JOURNAL OF HEMATOLOGY

Oral Iron Chelators

(2009) Maria Domenica Cappellini et al.   Annual Review of Medicine

Regulation of Hepcidin and Iron-Overload Disease

(2009) Pauline L. Lee et al.   Annual Review of Pathology-Mechanisms of Disease

Development of K562 cell clones expressing β-globin mRNA carrying the β039 thalassaemia mutation for the screening of correctors of stop-codon mutations

(2009) Francesca Salvatori et al.   BIOTECHNOLOGY AND APPLIED BIOCHEMISTRY

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells

(2009) Y.-J. Kim et al.   BLOOD

Amelioration of murine  -thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both  -globin and the MGMT drug-resistance gene

(2009) H. Zhao et al.   BLOOD

Identification of TWSG1 as a second novel erythroid regulator of hepcidin expression in murine and human cells

(2009) T. Tanno et al.   BLOOD

The HBS1L-MYB intergenic interval associated with elevated HbF levels shows characteristics of a distal regulatory region in erythroid cells

(2009) K. Wahlberg et al.   BLOOD

Umbilical cord blood transplantation: Pros, cons and beyond

(2009) Anfisa Stanevsky et al.   BLOOD REVIEWS

Parkinson's Disease Patient-Derived Induced Pluripotent Stem Cells Free of Viral Reprogramming Factors

(2009) Frank Soldner et al.   CELL

A Ferroportin Transcript that Lacks an Iron-Responsive Element Enables Duodenal and Erythroid Precursor Cells to Evade Translational Repression

(2009) De-Liang Zhang et al.   Cell Metabolism

Ineffective erythropoiesis and thalassemias

(2009) Stefano Rivella   CURRENT OPINION IN HEMATOLOGY

Genetic architecture of hemoglobin F control

(2009) Stephan Menzel et al.   CURRENT OPINION IN HEMATOLOGY

Survival after cord blood transplantation from unrelated donor as a second hematopoietic stem cell transplantation for recurrent pediatric acute myeloid leukemia

(2009) M. Oda et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

HIF-2α, but not HIF-1α, promotes iron absorption in mice

(2009) Maria Mastrogiannaki et al.   JOURNAL OF CLINICAL INVESTIGATION

Optimized Lentiviral Vector Design Improves Titer and Transgene Expression of Vectors Containing the Chicken β-Globin Locus HS4 Insulator Element

(2009) Hideki Hanawa et al.   MOLECULAR THERAPY

Developmental and species-divergent globin switching are driven by BCL11A

(2009) Vijay G. Sankaran et al.   NATURE

Virus-free induction of pluripotency and subsequent excision of reprogramming factors

(2009) Keisuke Kaji et al.   NATURE

piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells

(2009) Knut Woltjen et al.   NATURE

Genetic Variation on Chromosome 6 Influences F Cell Levels in Healthy Individuals of African Descent and HbF Levels in Sickle Cell Patients

(2009) Lisa E. Creary et al.   PLoS One

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice

(2009) S. Svasti et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hepcidin-induced internalization of ferroportin requires binding and cooperative interaction with Jak2

(2009) I. De Domenico et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases

(2009) L. Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reprogramming of murine fibroblasts to induced pluripotent stem cells with chemical complementation of Klf4

(2009) C. A. Lyssiotis et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Human Induced Pluripotent Stem Cells Free of Vector and Transgene Sequences

(2009) J. Yu et al.   SCIENCE

Stat5 regulates cellular iron uptake of erythroid cells via IRP-2 and TfR-1

(2008) M. A. Kerenyi et al.   BLOOD

Erythropoietin mediates hepcidin expression in hepatocytes through EPOR signaling and regulation of C/EBP 

(2008) J. P. Pinto et al.   BLOOD

Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in  -thalassemia

(2008) I. V. Libani et al.   BLOOD

Growth differentiation factor 15 production is necessary for normal erythroid differentiation and is increased in refractory anaemia with ring-sideroblasts

(2008) Jean-Marie Ramirez et al.   BRITISH JOURNAL OF HAEMATOLOGY

Regulation of Iron Absorption in Hemoglobinopathies

(2008) Gideon Rechavi et al.   CURRENT MOLECULAR MEDICINE

Serum hepcidin level and erythropoietic activity after hematopoietic stem cell transplantation

(2008) J. Kanda et al.   HAEMATOLOGICA

Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids

(2008) J. Y. Chin et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

Reprogramming of murine and human somatic cells using a single polycistronic vector

(2008) B. W. Carey et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

DNA polymorphisms at the BCL11A, HBS1L-MYB, and  -globin loci associate with fetal hemoglobin levels and pain crises in sickle cell disease

(2008) G. Lettre et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Induced Pluripotent Stem Cells Generated Without Viral Integration

(2008) M. Stadtfeld et al.   SCIENCE

Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A

(2008) Vijay G. Sankaran et al.   SCIENCE

Generation of Mouse Induced Pluripotent Stem Cells Without Viral Vectors

(2008) K. Okita et al.   SCIENCE

Induced Pluripotent Stem Cell Generation Using a Single Lentiviral Stem Cell Cassette

(2008) Cesar A. Sommer et al.   STEM CELLS

Fibroblast-Derived Induced Pluripotent Stem Cells Show No Common Retroviral Vector Insertions

(2008) Florencio Varas et al.   STEM CELLS

Promotion of Reprogramming to Ground State Pluripotency by Signal Inhibition

(2008) Jose Silva et al.   PLOS BIOLOGY