Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia (Q45888439)

17 December 2017

title

Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia (English)

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17 December 2017

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2

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17 December 2017

Roberto Gambari

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4

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17 December 2017

Laura Breda

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Laura Breda

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1

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Cristina Zuccato

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Cristina Zuccato

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3

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publication date

1 June 2013

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17 December 2017

Expert Review of Hematology

published in

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17 December 2017

volume

6

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17 December 2017

issue

3

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17 December 2017

page(s)

255-264

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17 December 2017

Molecular diagnosis of inherited disorders: lessons from hemoglobinopathies

cites work

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Screening and genetic diagnosis of haemoglobin disorders

21 January 2018

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21 January 2018

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Global burden, distribution and prevention of β-thalassemias and hemoglobin E disorders

21 January 2018

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Molecular therapies in beta-thalassaemia

21 January 2018

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An electronic infrastructure for research and treatment of the thalassemias and other hemoglobinopathies: the Euro-mediterranean ITHANET project

21 January 2018

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Medicinal chemistry of fetal hemoglobin inducers for treatment of beta-thalassemia

21 January 2018

1 reference

Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies.

21 January 2018

1 reference

Co-inheritance of alpha- and beta-thalassaemia in mice ameliorates thalassaemic phenotype.

21 January 2018

1 reference

Controlling alpha-globin: a review of alpha-globin expression and its impact on beta-thalassemia.

21 January 2018

1 reference

Gene therapy for hemoglobinopathies: progress and challenges

21 January 2018

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Fertility and pregnancy in thalassemia major

21 January 2018

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Growth and puberty and its management in thalassaemia

21 January 2018

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Complications of thalassemia major and their treatment

21 January 2018

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Alpha and beta cell evaluation in patients with thalassaemia intermedia and iron overload

21 January 2018

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Noninvasive assessment of liver fibrosis in thalassaemia major patients by transient elastography (TE) â lack of interference by iron deposition

21 January 2018

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Update on thalassemia: clinical care and complications

21 January 2018

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On the road to gene therapy for beta-thalassemia and sickle cell anemia

21 January 2018

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Current status of globin gene therapy for the treatment of beta-thalassaemia.

21 January 2018

1 reference

Gene therapy: Targeting β-thalassaemia

21 January 2018

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Hemoglobin gene therapy for β-thalassemia

21 January 2018

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Gene therapy for homozygous beta-thalassemia. Is it a reality?

21 January 2018

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Gene therapy for β-thalassaemia: the continuing challenge

21 January 2018

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Optimization of recombinant adeno-associated viral vectors for human beta-globin gene transfer and transgene expression.

21 January 2018

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Retroviral vectors for the beta-globin gene that demonstrate improved titer and expression

21 January 2018

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Evaluation of optimal expression cassette in retrovirus vector for beta-thalassemia gene therapy

21 January 2018

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Current advances in retroviral gene therapy

21 January 2018

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Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors

21 January 2018

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Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice.

21 January 2018

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Globin Lentiviral Vector Insertions Can Perturb the Expression of Endogenous Genes in β-thalassemic Hematopoietic Cells

21 January 2018

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Site-specific transfer of an intact beta-globin gene cluster through a new targeting vector.

21 January 2018

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In vivo selection of genetically modified erythroid cells using a jak2-based cell growth switch

21 January 2018

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The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in beta-thalassemia patients

21 January 2018

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Mobilization of hematopoietic stem cells in a thalassemic mouse model: implications for human gene therapy of thalassemia

21 January 2018

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Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease

21 January 2018

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Optimization of lentiviral vectors generation for biomedical and clinical research purposes: contemporary trends in technology development and applications

21 January 2018

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

21 January 2018

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Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

21 January 2018

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Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

21 January 2018

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High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells

21 January 2018

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Toward gene therapy for disorders of globin synthesis

21 January 2018

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The 3' region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity

21 January 2018

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A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia

21 January 2018

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A preclinical approach for gene therapy of beta-thalassemia.

21 January 2018

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

21 January 2018

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Expression of the human beta-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice

21 January 2018

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In vivo silencing of the human gamma-globin gene in murine erythroid cells following retroviral transduction

21 January 2018

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A 36-base-pair core sequence of locus control region enhances retrovirally transferred human beta-globin gene expression

21 January 2018

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Analysis of gamma-globin expression cassettes in retrovirus vectors

21 January 2018

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

21 January 2018

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Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human beta-globin in engrafted mice.

21 January 2018

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Fetal gene therapy of alpha-thalassemia in a mouse model

21 January 2018

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

21 January 2018

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Correction of sickle cell disease in transgenic mouse models by gene therapy

21 January 2018

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

21 January 2018

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The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene

21 January 2018

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Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients

21 January 2018

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Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region

21 January 2018

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The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites.

21 January 2018

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Genetic therapy for beta-thalassemia: from the bench to the bedside

21 January 2018

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

21 January 2018

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Gene therapy. Beta-thalassemia treatment succeeds, with a caveat

21 January 2018

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Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia

21 January 2018

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21 January 2018

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Recent advances in β-thalassemias

21 January 2018

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Fetal Hemoglobin Inducers from the Natural World: A Novel Approach for Identification of Drugs for the Treatment of {beta}-Thalassemia and Sickle-Cell Anemia.

21 January 2018

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A Novel Frameshift Mutation (+A) at Codon 18 of the β-Globin Gene Associated with High Persistence of Fetal Hemoglobin Phenotype and δβ-Thalassemia

21 January 2018

1 reference

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

21 January 2018

1 reference

Carrier screening for beta-thalassaemia: a review of international practice

21 January 2018

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Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells

21 January 2018

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

21 January 2018

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Response to hydroxyurea in beta thalassemia major and intermedia: experience in western India.

21 January 2018

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Hydroxyurea-induced hematological response in transfusion-independent beta-thalassemia intermedia: case series and review of literature

21 January 2018

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Thalidomide therapy in a patient with thalassemia major.

21 January 2018

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Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo

21 January 2018

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

21 January 2018

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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

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Partial correction of murine beta-thalassemia with a gammaretrovirus vector for human gamma-globin.

21 January 2018

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Effects of human gamma-globin in murine beta-thalassaemia

21 January 2018

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Discovering the genetics underlying foetal haemoglobin production in adults.

21 January 2018

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A functional element necessary for fetal hemoglobin silencing

21 January 2018

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Recent advances in the molecular understanding of non-transfusion-dependent thalassemia

21 January 2018

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Induction of endogenous gamma-globin gene expression with decoy oligonucleotide targeting Oct-1 transcription factor consensus sequence

21 January 2018

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

21 January 2018

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Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A.

21 January 2018

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Transcriptional silencing of fetal hemoglobin by BCL11A.

21 January 2018

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Targeted therapeutic strategies for fetal hemoglobin induction

21 January 2018

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MicroRNA-15a and -16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13

21 January 2018

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KLF1 regulates BCL11A expression and gamma- to beta-globin gene switching

21 January 2018

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Exploring strategies for the design of artificial transcription factors: targeting sites proximal to known regulatory regions for the induction of gamma-globin expression and the treatment of sickle cell disease

21 January 2018

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A zinc-finger transcriptional activator designed to interact with the gamma-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts

21 January 2018

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Recombinant erythroid Kruppel-like factor fused to GATA1 up-regulates delta- and gamma-globin expression in erythroid cells

21 January 2018

1 reference

Fetal hemoglobin levels and morbidity in untransfused patients with β-thalassemia intermedia.

21 January 2018

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A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction

21 January 2018

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Increase in gamma-globin mRNA content in human erythroid cells treated with angelicin analogs

21 January 2018

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Induction of human fetal hemoglobin via the NRF2 antioxidant response signaling pathway

21 January 2018

1 reference

A pilot study of subcutaneous decitabine in β-thalassemia intermedia

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1586/EHM.13.24

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17 December 2017

PubMed ID

23782080

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17 December 2017