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Quand l’ubiquitination se mêle du mélanome cutané: Un mécanisme à explorer

M.S. Médecine sciences, 2024-03, Vol.40 (3), p.308-310

2024

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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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Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression

Blood, 2017-12, Vol.130, p.5563-5563

2017

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Advances in the identification of deciduous molar tooth germs

Legal medicine (Tokyo, Japan), 2021-02, Vol.48, p.101801-101801, Article 101801

2021

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Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Blood, 2019-11, Vol.134 (Supplement_1), p.2242-2242

2019

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Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of γ-globin and β-globin gene promoters

Analytical and bioanalytical chemistry, 2019-11, Vol.411 (29), p.7669-7680

2019

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The challenging diagnosis of cranial congenital anomalies in a newborn from an Italian 20th century documented skeletal collection

International journal of osteoarchaeology, 2021-03, Vol.31 (2), p.309-315

2021

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A probable case of holoprosencephaly with cyclopia in a full-term fetus from a modern skeletal collection

International journal of paleopathology, 2021-06, Vol.33, p.25-29

2021

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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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Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

Molecular therapy, 2021-04, Vol.29 (4), p.1625-1638

2021

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Improved Lentiviral Vectors for the Cure of Hemoglobinopathies

Blood, 2018-11, Vol.132 (Supplement 1), p.3477-3477

2018

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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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A validated cellular biobank for β-thalassemia

Journal of translational medicine, 2016-09, Vol.14 (1), p.255-255, Article 255

2016

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Lack of GDF11 Does Not Ameliorate Erythropoiesis in β-Thalassemia and Does Not Prevent the Activity of the Trap-Ligand RAP-536

Blood, 2018-11, Vol.132 (Supplement 1), p.165-165

2018

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2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood, 2019-11, Vol.134 (Supplement_1), p.2244-2244

2019

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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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Comparing Strategies to Reactivate Fetal Globin Expression for the Treatment of Beta-Globinopathies

Blood, 2014-12, Vol.124 (21), p.333-333

2014

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Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS

Blood, 2016-12, Vol.128 (22), p.2439-2439

2016

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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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Macrophages Regulate Stress Erythropoiesis Through Direct Cellular Interactions Associated With Integrin β1-Focal Adhesion Kinase Signaling

Blood, 2013-11, Vol.122 (21), p.307-307

2013

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Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos

Blood, 2016-12, Vol.128 (22), p.1014-1014

2016

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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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Quand l’ubiquitination se mêle du mélanome cutané: Un mécanisme à explorer

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

Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Advances in the identification of deciduous molar tooth germs

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of γ-globin and β-globin gene promoters

The challenging diagnosis of cranial congenital anomalies in a newborn from an Italian 20th century documented skeletal collection

A probable case of holoprosencephaly with cyclopia in a full-term fetus from a modern skeletal collection

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

Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

Improved Lentiviral Vectors for the Cure of Hemoglobinopathies

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

A validated cellular biobank for β-thalassemia

Lack of GDF11 Does Not Ameliorate Erythropoiesis in β-Thalassemia and Does Not Prevent the Activity of the Trap-Ligand RAP-536

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

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

Comparing Strategies to Reactivate Fetal Globin Expression for the Treatment of Beta-Globinopathies

Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS

Recent trends in the gene therapy of β-thalassemia

Macrophages Regulate Stress Erythropoiesis Through Direct Cellular Interactions Associated With Integrin β1-Focal Adhesion Kinase Signaling

Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos

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

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