Resources

Human genetic and immunological determinants of critical COVID-19 pneumonia.

Qian Zhang, Paul Bastard, Aurélie Cobat, Jean-Laurent CASANOVA , Anna-Lena Neehus, Anne Puel, Bertrand Boisson, Daniela Matuozzo, Emmanuelle Jouanguy, Jean-Laurent CASANOVA , Jérémie Rosain, Jeremy Manry, Laurent Abel , Lucia Victoria Erazo, Quentin Philippot, Romain Levy, Shen-Ying Zhang, Stéphanie Boisson-Dupuis, Tom Le Voyer, Vivien Beziat

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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.

Anne Chalumeau, Adeline DENIS, Bénédicte Neven, Emmanuelle SIX, Annarita Miccio , Marina Cavazzana

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Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.

Annarita Miccio , Panagiotis Antoniou, Sorana CIURA, Edor Kabashi

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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.

Sophie Ramadier, Anne Chalumeau, Tristan Felix, Giacomo Frati, Mégane Brusson, Marina Cavazzana, Vasco Meneghini, Annarita Miccio

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Dystrophic epidermolysis bullosa pruriginosa: a new case series of a rare phenotype unveils skewed Th2 immunity.

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Trial of Spesolimab for Generalized Pustular Psoriasis.

Hervé Bachelez

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Trial of Spesolimab for Generalized Pustular Psoriasis.

Hervé Bachelez

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Drug Repurposing Reveals mTOR Inhibition as a Promising Strategy for Epidermolysis Bullosa Simplex.

Hélène Ragot, Alain Hovnanian

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A CBF decrease in the left supplementary motor areas: New insight into postoperative pediatric cerebellar mutism syndrome using arterial spin labeling perfusion MRI.

Jennifer Boisgontier, Ludovic Fillon, Caroline Rutten , Ana Saitovitch, Hervé Lemaître, Thomas Blauwblomme, Alice Vinçon-Leite, Monica Zilbovicius , Nathalie Boddaert

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Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced dysfunction.

Baptiste LAMARTHÉE, Soëli CHARBONNIER, Tifanie BLEIN, Emmanuel Martin, Matthias Titeux, Marianne DELVILLE, Hélène VINÇON, Emmanuelle SIX, Sylvain Latour, Marina Cavazzana, Isabelle André, Julien ZUBER

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Pustular psoriasis: Molecular pathways and effects of spesolimab in generalized pustular psoriasis.

Hervé Bachelez

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Enhanced cGAS-STING-dependent interferon signaling associated with mutations in ATAD3A.

Alice Lepelley, Marie-Louise Frémond, Luís Seabra, Yanick Crow

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Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease.

Michael Dussiot, Giacomo Frati, Annarita Miccio

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Immunity to EBV as revealed by immunedeficiencies.

Sylvain Latour

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SARS-CoV-2 infection inducing severe flare up of Deficiency of Interleukin Thirty-six (IL-36) Receptor Antagonist (DITRA) resulting from a mutation invalidating the activating cleavage site of the IL-36 receptor antagonist.

Hervé Bachelez

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