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Reactivation of Developmentally Silenced Globin Genes by Forced Chromatin Looping

Cell, 2014-08, Vol.158 (4), p.849-860

2014

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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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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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Modulators of Erythropoiesis

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

2014

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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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In vivo hematopoietic stem cell modification by mRNA delivery

Science (American Association for the Advancement of Science), 2023-07, Vol.381 (6656), p.436-443

2023

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Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy

Blood, 2021-11, Vol.138 (Supplement 1), p.3131-3131

2021

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Gene therapy for hemoglobinopathies: progress and challenges

Translational research : the journal of laboratory and clinical medicine, 2013-04, Vol.161 (4), p.293-306

2013

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AMPK Regulates the Expression of the Fe-S Cluster Assembly Enzyme (ISCU) and ALAS2, Modulating Cellular Iron Metabolism and Increasing Hemoglobin Synthesis

Blood, 2018-11, Vol.132 (Supplement 1), p.851-851

2018

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Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting β-Thalassemia

Blood, 2022-11, Vol.140 (Supplement 1), p.1944-1945

2022

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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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Identification and Characterization of Small Molecules That Inhibit Nonsense-Mediated RNA Decay and Suppress Nonsense p53 Mutations

Cancer research (Chicago, Ill.), 2014-06, Vol.74 (11), p.3104-3113

2014

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Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

Molecular diagnosis & therapy, 2019-04, Vol.23 (2), p.173-186

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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Elevated P21 (CDKN1a) Mediates Apoptosis of Beta-Thalassemic Erythroid Cells in Mice but Its Ablation Doesn't Improve Erythroid Maturation

Blood, 2020-11, Vol.136 (Supplement 1), p.19-19

2020

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Combination of a Luspatercept-like Drug (RAP-GRL) and Tmprss6-ASO Is Superior to Either Drug Alone for Correcting β-Thalassemia

Blood, 2021-11, Vol.138 (Supplement 1), p.2013-2013

2021

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In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA

Blood, 2022-11, Vol.140 (Supplement 1), p.305-306

2022

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Macrophages support pathological erythropoiesis in polycythemia vera and β-thalassemia

Nature medicine, 2013-04, Vol.19 (4), p.437

2013

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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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Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of [gamma]-globin and [beta]-globin gene promoters

Analytical and bioanalytical chemistry, 2019-11, Vol.411 (29), p.7669

2019

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A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

Blood, 2021-11, Vol.138 (Supplement 1), p.2012-2012

2021

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Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia

Blood, 2022-11, Vol.140 (Supplement 1), p.300-302

2022

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Improved Gene Therapy for Metachromatic Leukodystrophy

Blood, 2021-11, Vol.138 (Supplement 1), p.3979-3979

2021

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Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice

The Journal of clinical investigation, 2010-12, Vol.120 (12), p.4466-4477

2010

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Reactivation of Developmentally Silenced Globin Genes by Forced Chromatin Looping

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

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

Modulators of Erythropoiesis

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

In vivo hematopoietic stem cell modification by mRNA delivery

Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy

Gene therapy for hemoglobinopathies: progress and challenges

AMPK Regulates the Expression of the Fe-S Cluster Assembly Enzyme (ISCU) and ALAS2, Modulating Cellular Iron Metabolism and Increasing Hemoglobin Synthesis

Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting β-Thalassemia

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

Identification and Characterization of Small Molecules That Inhibit Nonsense-Mediated RNA Decay and Suppress Nonsense p53 Mutations

Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

Gene therapy in thalassemia and hemoglobinopathies

Elevated P21 (CDKN1a) Mediates Apoptosis of Beta-Thalassemic Erythroid Cells in Mice but Its Ablation Doesn't Improve Erythroid Maturation

Combination of a Luspatercept-like Drug (RAP-GRL) and Tmprss6-ASO Is Superior to Either Drug Alone for Correcting β-Thalassemia

In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA

Macrophages support pathological erythropoiesis in polycythemia vera and β-thalassemia

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

Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of [gamma]-globin and [beta]-globin gene promoters

A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia

Improved Gene Therapy for Metachromatic Leukodystrophy

Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice

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