Présentation
Publications scientifiques
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2023
Rescuing the cytolytic function of APDS1 patient T cells via TALEN-mediated P..., Mol Ther Methods Clin Dev
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2023
Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry..., J Allergy Clin Immunol
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2023
Novel lentiviral vectors for gene therapy of sickle cell disease combining ge..., Mol Ther Nucleic Acids
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2023
Successful treatment of severe MSUD in Bckdhb mice with neonatal AAV gene the..., J Inherit Metab Dis
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2021
A DL-4- and TNFα-based culture system to generate high numbers of nonmodified..., Cell Mol Immunol
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2021
A gain-of-function RAC2 mutation is associated with bone-marrow hypoplasia an..., Haematologica
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2020
Successful Preclinical Development of Gene Therapy for Recombinase-Activating..., Mol Ther Methods Clin Dev
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2020
Donor-targeted serotherapy as a rescue therapy for steroid-resistant acute GV..., Am J Transplant
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2019
Extensive multilineage analysis in patients with mixed chimerism after alloge..., Haematologica
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2019
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene T..., Mol Ther Methods Clin Dev
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2019
Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune..., Bone Marrow Transplant
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2019
Gene therapy of hemoglobinopathies: progress and future challenges., Hum Mol Genet
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2019
Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of ..., Hum Gene Ther
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2019
Gene therapy targeting haematopoietic stem cells for inherited diseases: prog..., Nat Rev Drug Discov
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2019
Loss of ARHGEF1 causes a human primary antibody deficiency., J Clin Invest
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2018
A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduc..., Mol Ther Methods Clin Dev
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2018
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Dis..., Mol Ther Methods Clin Dev
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2018
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-K..., Front Immunol
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2018
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem ..., Haematologica
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2017
Generation of adult human T-cell progenitors for immunotherapeutic applications., J Allergy Clin Immunol
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2017
Mutations in the adaptor-binding domain and associated linker region of p110δ..., Haematologica
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2016
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point ..., Stem Cells Dev
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2016
X-linked primary immunodeficiency associated with hemizygous mutations in the..., J Allergy Clin Immunol
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2016
Clinical and immunologic phenotype associated with activated phosphoinositide..., J Allergy Clin Immunol
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2016
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?, Hum Gene Ther
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2015
AK2 deficiency compromises the mitochondrial energy metabolism required for d..., Cell Death Dis
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2015
An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, ..., J Allergy Clin Immunol
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2015
A human immunodeficiency caused by mutations in the PIK3R1 gene., J Clin Invest
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2014
Successful RAG1-SCID gene therapy depends on the level of RAG1 expression., J Allergy Clin Immunol
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2014
The BLNK adaptor protein has a nonredundant role in human B-cell differentiat..., J Allergy Clin Immunol
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2013
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined im..., J Allergy Clin Immunol