E
Emmanuel Payen
Researcher at French Institute of Health and Medical Research
Publications - 46
Citations - 5516
Emmanuel Payen is an academic researcher from French Institute of Health and Medical Research. The author has contributed to research in topics: Genetic enhancement & Stem cell. The author has an hindex of 23, co-authored 44 publications receiving 4883 citations. Previous affiliations of Emmanuel Payen include University of Paris-Sud & Commissariat à l'énergie atomique et aux énergies alternatives.
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Journal ArticleDOI
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier,Salima Hacein-Bey-Abina,Cynthia C. Bartholomae,Gabor Istvan Veres,Manfred Schmidt,Ina Kutschera,Michel Vidaud,Ulrich Abel,Liliane Dal-Cortivo,Laure Caccavelli,Nizar Mahlaoui,Veronique Kiermer,Denice Mittelstaedt,Céline Bellesme,Najiba Lahlou,François Lefrère,Stéphane Blanche,Muriel Audit,Emmanuel Payen,Philippe Leboulch,Philippe Leboulch,Bruno l’Homme,Pierre Bougnères,Christof von Kalle,Alain Fischer,Marina Cavazzana-Calvo,Patrick Aubourg +26 more
TL;DR: Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.
Journal ArticleDOI
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo,Emmanuel Payen,Olivier Negre,Gary P. Wang,Kathleen M. Hehir,Floriane Fusil,Julian D. Down,Maria Denaro,Troy Brady,Karen A. Westerman,Resy Cavallesco,Beatrix Gillet-Legrand,Laure Caccavelli,Laure Caccavelli,Riccardo Sgarra,Leila Maouche-Chretien,Françoise Bernaudin,Robert Girot,Ronald Dorazio,Geert Jan Mulder,Axel Polack,Arthur Bank,Jean Soulier,Jérôme Larghero,Nabil Kabbara,Bruno Dalle,Bernard Gourmel,Gérard Socié,Stany Chrétien,Stany Chrétien,Nathalie Cartier,Patrick Aubourg,Alain Fischer,Alain Fischer,Kenneth Cornetta,Frédéric Galactéros,Yves Beuzard,Eliane Gluckman,Frederick D. Bushman,Salima Hacein-Bey-Abina,Salima Hacein-Bey-Abina,Philippe Leboulch +41 more
TL;DR: It is shown that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.
Journal ArticleDOI
Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy
Robert Pawliuk,Karen A. Westerman,Mary E. Fabry,Emmanuel Payen,Robert Tighe,Eric E. Bouhassira,Seetharama A. Acharya,James Ellis,Irving M. London,Connie J. Eaves,R. Keith Humphries,Yves Beuzard,Ronald L. Nagel,Philippe Leboulch +13 more
TL;DR: In two mouse SCD models, Berkeley and SAD, inhibition of red blood cell dehydration and sickling was achieved with correction of hematological parameters, splenomegaly, and prevention of the characteristic urine concentration defect.
Journal ArticleDOI
Gene Therapy in a Patient with Sickle Cell Disease.
Jean-Antoine Ribeil,Salima Hacein-Bey-Abina,Emmanuel Payen,Alessandra Magnani,Michaela Semeraro,Elisa Magrin,Laure Caccavelli,Bénédicte Neven,Philippe Bourget,Wassim El Nemer,Pablo Bartolucci,Leslie Weber,Hervé Puy,Jean-François Meritet,David Grevent,Yves Beuzard,Stany Chrétien,Thibaud Lefebvre,Robert W. Ross,Olivier Negre,Gabor Istvan Veres,Laura Sandler,Sandeep Soni,Mariane de Montalembert,Stéphane Blanche,Philippe Leboulch,Marina Cavazzana +26 more
TL;DR: First patient treated with lentiviral vector–mediated addition of an antisickling β‐globin gene into autologous hematopoietic stem cells without recurrence of sickle crises and with correction of the biologic hallmarks of the disease is described.
Journal ArticleDOI
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Alexis A. Thompson,Mark C. Walters,Janet L. Kwiatkowski,John E.J. Rasko,John E.J. Rasko,John E.J. Rasko,Jean-Antoine Ribeil,Suradej Hongeng,Elisa Magrin,Gary J. Schiller,Emmanuel Payen,Michaela Semeraro,Despina Moshous,François Lefrère,Hervé Puy,Philippe Bourget,Alessandra Magnani,Laure Caccavelli,Jean-Sebastien Diana,Felipe Suarez,Fabrice Monpoux,Valentine Brousse,Catherine Poirot,Catherine Poirot,Chantal Brouzes,Jean-François Meritet,Corinne Pondarré,Yves Beuzard,Stany Chrétien,Thibaud Lefebvre,David T. Teachey,Usanarat Anurathapan,P. Joy Ho,P. Joy Ho,Christof von Kalle,Morris Kletzel,Elliott Vichinsky,Sandeep Soni,Gabor Istvan Veres,Olivier Negre,Robert W. Ross,David Davidson,Alexandria Petrusich,Laura Sandler,Mohammed Asmal,Olivier Hermine,Mariane de Montalembert,Salima Hacein-Bey-Abina,Stéphane Blanche,Philippe Leboulch,Philippe Leboulch,Philippe Leboulch,Marina Cavazzana +52 more
TL;DR: Gene therapy with autologous CD34+ cells transduced with the BB305 vector reduced or eliminated the need for long‐term red‐cell transfusions in 22 patients with severe β‐thalassemia without serious adverse events related to the drug product.