Publications and ressources

Single-cell analysis of FOXP3 deficiencies in humans and mice unmasks intrinsic and extrinsic CD4+ T cell perturbations.

Emmanuelle SIX, Marianne DELVILLE, Julien ZUBER, Bénédicte Neven, Nadine CERF-BENSUSSAN , Frédéric Rieux-Laucat, Marina Cavazzana, Isabelle André

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A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome.

Marianne DELVILLE, Florence BELLIER, Sabrina LIZOT, Hélène VINÇON, Steicy SOBRINO, Romane THOUENON, Alexandrine GARRIGUE, Juliette OLIVRÉ, Soëli CHARBONNIER, Chantal LAGRESLE-PEYROU, Baptiste LAMARTHÉE, Julien ZUBER, Isabelle André, Marina Cavazzana, Emmanuelle SIX

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Adenylate kinase 2 expression and addiction in T-ALL.

Emmanuelle SIX, Chantal LAGRESLE-PEYROU

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Improving the diagnostic efficiency of primary immunodeficiencies with targeted next-generation sequencing.

Jérémie Rosain, Brigitte Bader-Meunier, Olivier Hermine, Felipe Suarez, Isabelle André, Vivien Beziat, Geneviève de Saint Basile, Jean-Pierre De Villartay, Sven KRACKER, Chantal LAGRESLE-PEYROU, Sylvain Latour, Frédéric Rieux-Laucat, Christine Bole, Patrick Nitschké, Alain Fischer, Despina Moshous, Bénédicte Neven, Alexandre Alcaïs, Jacinta Bustamante

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Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies.

Despina Moshous, Felipe Suarez, Marina Cavazzana, Sven KRACKER

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Donor-targeted serotherapy as a rescue therapy for steroid-resistant acute GVHD after HLA-mismatched kidney transplantation.

Julien ZUBER, Olivia Boyer, Bénédicte Neven, Romain Levy, Baptiste LAMARTHÉE, Michael Dussiot, Isabelle André, Marina Cavazzana

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From Dysgammaglobulinemia to Autosomal-Dominant Activation-Induced Cytidine Deaminase Deficiency: Unraveling an Inherited Immunodeficiency after 50 Years.

Loïc CHENTOUT, Bertrand Boisson, Aurore POULIET, Anne DURANDY, Jean-Laurent CASANOVA , Sven KRACKER

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Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.

Chantal LAGRESLE-PEYROU, Marina Cavazzana

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From Dysgammaglobulinemia to Autosomal-Dominant Activation-Induced Cytidine Deaminase Deficiency: Unraveling an Inherited Immunodeficiency after 50 Years.

Loïc CHENTOUT, Bertrand Boisson, Aurore POULIET, Anne DURANDY, Jean-Laurent CASANOVA , Sven KRACKER

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Topoisomerase 2β mutation impairs early B-cell development.

Olivier Hermine, Alain Fischer, Anne DURANDY, Sven KRACKER

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NHP2 deficiency impairs rRNA biogenesis and causes pulmonary fibrosis and Høyeraal-Hreidarsson syndrome.

Laëtitia Kermasson, Chantal LAGRESLE-PEYROU, Emmanuelle Ollivier, Alicia Fernandes, Jean-Pierre De Villartay, Patrick Revy

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Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Emmanuelle SIX, Adeline DENIS, Marianne DELVILLE, Steicy SOBRINO, Isabelle André, Marina Cavazzana

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A gain-of-function RAC2 mutation is associated with bone-marrow hypoplasia and an autosomal dominant form of severe combined immunodeficiency.

Chantal LAGRESLE-PEYROU, Hanem SADEK, Cindy DA SILVA, Alexandrine GARRIGUE, Isabelle André, Marina Cavazzana

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Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.

Chantal LAGRESLE-PEYROU, Alain Fischer, Jean-Pierre De Villartay, Marina Cavazzana

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A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells.

Marianne DELVILLE, Adeline DENIS, Chantal LAGRESLE-PEYROU, Marina Cavazzana, Isabelle André, Emmanuelle SIX

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