Publications and ressources

Pseudodynamic analysis of heart tube formation in the mouse reveals strong regional variability and early left-right asymmetry.

Audrey Desgrange, Sigolène Meilhac

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Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

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Shaping the mouse heart tube from the second heart field epithelium.

Paul Palmquist-Gomes, Sigolène Meilhac

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De novo generation of the NPM-ALK fusion recapitulates the pleiotropic phenotypes of ALK+ ALCL pathogenesis and reveals the ROR2 receptor as target for tumor cells.

Loelia Babin, Alice Darchen, Rosalie Borry, Claire Soudais, Lucile Couronné, Erika Brunet

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Targeted next-generation sequencing in a large series of fetuses with severe renal diseases.

Guillaume Dorval, Christelle Arrondel, Tania Attié-Bitach, Cécile Jeanpierre, Sophie Saunier, Laurence Heidet

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

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3D cell morphology detection by association for embryo heart morphogenesis.

Daniel Darby, Sigolène Meilhac

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Periodic electroencephalographic discharges and epileptic spasms involve cortico-striatal-thalamic loops on Arterial Spin Labeling Magnetic Resonance Imaging.

Ludovic Fillon, Ana Saitovitch, Jennifer Boisgontier, Alice Vinçon-Leite, Thomas Blauwblomme, Monica Zilbovicius , Rima NABBOUT, Nathalie Boddaert

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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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