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CURING HEMOGLOBINOPATHIES: CHALLENGES AND ADVANCES OF CONVENTIONAL AND NEW GENE THERAPY APPROACHES

Mediterranean journal of hematology and infectious diseases, 2019-11, Vol.11 (1), p.e2019067-e2019067

2019

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Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin

Haematologica (Roma), 2021-10, Vol.106 (10), p.2740-2745

2021

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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

PloS one, 2012-03, Vol.7 (3), p.e32345-e32345

2012

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Downregulation of hepcidin and haemojuvelin expression in the hepatocyte cell‐line HepG2 induced by thalassaemic sera

British journal of haematology, 2006-10, Vol.135 (1), p.129-138

2006

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Modulators of erythropoiesis: emerging therapies for hemoglobinopathies and disorders of red cell production

Hematology/oncology clinics of North America, 2014-04, Vol.28 (2), p.375-386

2014

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Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia

Blood, 2019-08, Vol.134 (6), p.568-572

2019

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Gene therapy in thalassemia and hemoglobinopathies

Mediterranean journal of hematology and infectious diseases, 2009-11, Vol.1 (1), p.e2009008-e2009008

2009

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Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

Blood, 2016-08, Vol.128 (8), p.1139-1143

2016

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Elevated CDKN1a (p21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesis

Blood advances, 2023-11, Vol.7 (22), p.6873-6885

2023

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Ineffective erythropoiesis in β-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin

Blood, 2007-06, Vol.109 (11), p.5027-5035

2007

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Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in β-thalassemia

Blood, 2008-08, Vol.112 (3), p.875-885

2008

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Recent trends in the gene therapy of β-thalassemia

Journal of Blood Medicine, 2015-01, Vol.6 (default), p.69-85

2015

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

Expert review of hematology, 2013-06, Vol.6 (3), p.255-264

2013

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A preclinical approach for gene therapy of β-thalassemia

Annals of the New York Academy of Sciences, 2010-08, Vol.1202 (1), p.134-140

2010

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Decreased hepcidin mRNA expression in thalassemic mice

British journal of haematology, 2004-01, Vol.124 (1), p.123-124

2004

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mRNA expression of iron regulatory genes in β‐thalassemia intermedia and β‐thalassemia major mouse models

American journal of hematology, 2006-07, Vol.81 (7), p.479-483

2006

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

Annals of hematology, 2012-08, Vol.91 (8), p.1201-1213

2012

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Production of beta-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous beta(0)39 thalassemia patients

American journal of hematology, 2009-11, Vol.84 (11), p.720-728

2009

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Exploring the Role of Hepcidin, an Antimicrobial and Iron Regulatory Peptide, in Increased Iron Absorption in β-Thalassemia

Annals of the New York Academy of Sciences, 2005-11, Vol.1054 (1), p.417-422

2005

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Oral mucositis assessment in pediatric and adolescent oncological patients: A systematic review

Pediatric blood & cancer, 2024-10, p.e31388

2024

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Carotid Intima-Media Thickness: Novel Loci, Sex-Specific Effects, and Genetic Correlations With Obesity and Glucometabolic Traits in UK Biobank

Arteriosclerosis, thrombosis, and vascular biology, 2020-02, Vol.40 (2), p.446-461

2020

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CURING HEMOGLOBINOPATHIES: CHALLENGES AND ADVANCES OF CONVENTIONAL AND NEW GENE THERAPY APPROACHES

Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin

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

Downregulation of hepcidin and haemojuvelin expression in the hepatocyte cell‐line HepG2 induced by thalassaemic sera

Modulators of erythropoiesis: emerging therapies for hemoglobinopathies and disorders of red cell production

Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia

Gene therapy in thalassemia and hemoglobinopathies

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

Elevated CDKN1a (p21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesis

Ineffective erythropoiesis in β-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin

Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in β-thalassemia

Recent trends in the gene therapy of β-thalassemia

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

A preclinical approach for gene therapy of β-thalassemia

Decreased hepcidin mRNA expression in thalassemic mice

mRNA expression of iron regulatory genes in β‐thalassemia intermedia and β‐thalassemia major mouse models

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

Production of beta-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous beta(0)39 thalassemia patients

Exploring the Role of Hepcidin, an Antimicrobial and Iron Regulatory Peptide, in Increased Iron Absorption in β-Thalassemia

Oral mucositis assessment in pediatric and adolescent oncological patients: A systematic review

Carotid Intima-Media Thickness: Novel Loci, Sex-Specific Effects, and Genetic Correlations With Obesity and Glucometabolic Traits in UK Biobank

Polygenic risk of major depressive disorder as a risk factor for venous thromboembolism

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