Molecular therapies in beta-thalassaemia (Q36665342)

15 August 2017

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title

Molecular therapies in beta-thalassaemia (English)

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15 August 2017

2

Swee Lay Thein

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15 August 2017

Lynn Quek

1

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PubMed ID

17129232

15 August 2017

language of work or name

English

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publication date

27 November 2006

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British Journal of Haematology

15 August 2017

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15 August 2017

136

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15 August 2017

3

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15 August 2017

353-365

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Flow cytometric analysis of the oxidative status of normal and thalassemic red blood cells

15 August 2017

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Long-term survival of ex-thalassemic patients with persistent mixed chimerism after bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Short-chain fatty acid derivatives stimulate cell proliferation and induce STAT-5 activation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Hydroxyurea can eliminate transfusion requirements in children with severe β-thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Correction of the sickle cell mutation in embryonic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Binding of HMG-I(Y) elicits structural changes in a silencer of the human beta-globin gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Oxidative status and malondialdehyde in beta-thalassaemia patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Hydroxyurea induces fetal hemoglobin by the nitric oxide-dependent activation of soluble guanylyl cyclase

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Maintenance of elevated fetal hemoglobin levels by decitabine during dose interval treatment of sickle cell anemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Umbilical cord blood transplantation in Chinese children with beta-thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Use of the hereditary persistence of fetal hemoglobin 2 enhancer to increase the expression of oncoretrovirus vectors for human gamma-globin

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Somatic deletion of the normal beta-globin gene leading to thalassaemia intermedia in heterozygous beta-thalassaemic patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Stem cell transplantation for hemoglobinopathies

21 January 2018

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https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Bone marrow transplantation in adults with thalassemia: Treatment and long-term follow-up

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Biophysical characterization of the alpha-globin binding protein alpha-hemoglobin stabilizing protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Balancing acts: molecular control of mammalian iron metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

cMYB is involved in the regulation of fetal hemoglobin production in adults.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

A rare example that coinheritance of a severe form of beta-thalassemia and alpha-thalassemia interact in a "synergistic" manner to balance the phenotype of classic thalassemic syndromes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

An abundant erythroid protein that stabilizes free alpha-haemoglobin

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Alpha-haemoglobin stabilising protein is a quantitative trait gene that modifies the phenotype of beta-thalassaemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Hydroxyurea therapy in thalassemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Gene Therapy for beta-thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Short-chain fatty acids induce gamma-globin gene expression by displacement of a HDAC3-NCoR repressor complex

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Short-chain fatty acid derivatives induce fetal globin expression and erythropoiesis in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Fetal globin induction--can it cure beta thalassemia?

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

The role of recombinant human erythropoietin in the treatment of thalassemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Synthetic hepcidin causes rapid dose-dependent hypoferremia and is concentrated in ferroportin-containing organs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Murine and math models for the level of stable mixed chimerism to cure beta-thalassemia by nonmyeloablative bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Pathophysiology of alpha- and beta-thalassemia: therapeutic implications

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

A minimal ankyrin promoter linked to a human gamma-globin gene demonstrates erythroid specific copy number dependent expression with minimal position or enhancer dependence in transgenic mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Regression of extramedullary haemopoiesis and augmentation of fetal haemoglobin concentration during hydroxyurea therapy in beta thalassaemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Pathophysiology of thalassemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Control of globin gene expression during development and erythroid differentiation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Genetic insights into the clinical diversity of beta thalassaemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Pathophysiology of beta thalassemia--a guide to molecular therapies

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Mutations in the general transcription factor TFIIH result in beta-thalassaemia in individuals with trichothiodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Evaluation of alpha hemoglobin stabilizing protein (AHSP) as a genetic modifier in patients with beta thalassemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Thalassemia--a global public health problem

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Correction of sickle cell disease by homologous recombination in embryonic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Activation of human gamma-globin gene expression via triplex-forming oligonucleotide (TFO)-directed mutations in the gamma-globin gene 5' flanking region

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

X-linked thrombocytopenia with thalassemia from a mutation in the amino finger of GATA-1 affecting DNA binding rather than FOG-1 interaction

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

Inhibition of beta protein 1 expression enhances beta-globin promoter activity and beta-globin mRNA levels in the human erythroleukemia (K562) cell line

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

21 January 2018

Haemoglobin's chaperone

1 reference

https://api.crossref.org/works/10.1111%2FJ.1365-2141.2006.06408.X

10.1111/J.1365-2141.2006.06408.X

21 January 2018

Identifiers

DOI

1 reference

15 August 2017

1 reference

PubMed ID

17129232