World's Best Scientists 2026 revealed!

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Molecular Biology

D-Index
56
Citations
18057
World Ranking
2183
National Ranking
1083

Overview

Philippe Leboulch is affiliated with Brigham and Women's Hospital in the United States. Their research focuses primarily on Medicine and Biochemistry, Genetics and Molecular Biology, with significant contributions to the fields of Genetics, Molecular Biology, Pediatrics, Perinatology and Child Health, Infectious Diseases, and Hematology.

The scientist's main research topics include:

  • Hemoglobinopathies and Related Disorders
  • Prenatal Screening and Diagnostics
  • Parvovirus B19 Infection Studies
  • Virus-based gene therapy research
  • Iron Metabolism and Disorders
  • Pluripotent Stem Cells Research
  • Liver physiology and pathology

Leboulch has published in various journals and scientific venues, notably including:

  • Nature Medicine
  • American Journal of Hematology
  • Molecular Therapy
  • International Journal of Molecular Sciences
  • Cytotherapy

Recent publications demonstrate a focus on gene therapy and hematologic diseases. Selected recent papers include:

  • "Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial" (2022, Nature Medicine)
  • "Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study" (2022, American Journal of Hematology)
  • "Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia" (2021, Molecular Therapy)
  • "Successful Derivation of Hepatoblasts, Cholangiocytes and Hepatocytes from Simian Induced Pluripotent Stem Cells" (2022, International Journal of Molecular Sciences)
  • "Droplet digital polymerase chain reaction-based quantitation of therapeutic lentiviral vector copies in transduced hematopoietic stem cells" (2024, Cytotherapy)

The scientist frequently collaborates with the following researchers:

  • Emmanuel Payen (3 joint publications)
  • Mohammed Asmal (2 joint publications)
  • Manfred Schmidt (2 joint publications)
  • Jean-Antoine Ribeil (2 joint publications)
  • Suradej Hongeng (2 joint publications)

Best Publications

  • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

    S. Hacein-Bey-Abina;C. Von Kalle;C. Von Kalle;M. Schmidt;M. P. McCormack

  • Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

    Nathalie Cartier;Salima Hacein-Bey-Abina;Cynthia C. Bartholomae;Gabor Veres

  • Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

    Marina Cavazzana-Calvo;Emmanuel Payen;Olivier Negre;Gary Wang

  • Gene Therapy in a Patient with Sickle Cell Disease.

    Jean-Antoine Ribeil;Salima Hacein-Bey-Abina;Emmanuel Payen;Alessandra Magnani

  • Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

    Alexis A. Thompson;Mark C. Walters;Janet Kwiatkowski;John E.J. Rasko;John E.J. Rasko;John E.J. Rasko

  • Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy

    Robert Pawliuk;Karen A. Westerman;Mary E. Fabry;Emmanuel Payen

  • Heme-regulated eIF2α kinase (HRI) is required for translational regulation and survival of erythroid precursors in iron deficiency

    An-Ping Han;Channing Yu;Linrong Lu;Yuko Fujiwara

  • Eukaryotic Translation Initiation Factor 4E Regulates Expression of Cyclin D1 at Transcriptional and Post-transcriptional Levels

    Igor B. Rosenwald;Roger Kaspar;Dennis Rousseau;Lee Gehrke

  • Erosion of the chronic myeloid leukaemia stem cell pool by PPARγ agonists.

    Stéphane Prost;Francis Relouzat;Marc Spentchian;Yasmine Ouzegdouh

  • Site-specific chromosomal integration in mammalian cells: highly efficient CRE recombinase-mediated cassette exchange.

    Yong Qing Feng;Jost Seibler;Raouf Alami;Andrew Eisen

  • Reversible immortalization of mammalian cells mediated by retroviral transfer and site-specific recombination

    Karen A. Westerman;Philippe Leboulch

  • Placenta Growth Factor-1 antagonizes VEGF-induced angiogenesis and tumor growth by the formation of functionally inactive PlGF-1/VEGF heterodimers

    Anna Eriksson;Renhai Cao;Robert Pawliuk;Sanna Maria Berg

  • Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure

    P. Leboulch;G. M. S. Huang;R. K. Humphries;Y. H. Oh

  • A phase I/II clinical trial of β-globin gene therapy for β-thalassemia

    Arthur Bank;Ronald Dorazio;Philippe Leboulch

  • Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells

    Suzan Imren;Emmanuel Payen;Karen A. Westerman;Robert Pawliuk

  • A human β-cell line for transplantation therapy to control type 1 diabetes

    Michiki Narushima;Naoya Kobayashi;Teru Okitsu;Yoshihito Tanaka

  • Transcriptional Behavior of LCR Enhancer Elements Integrated at the Same Chromosomal Locus by Recombinase-Mediated Cassette Exchange

    Eric E. Bouhassira;Karen Westerman;Karen Westerman;Karen Westerman;Philippe Leboulch;Philippe Leboulch;Philippe Leboulch

  • Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene

    Olivier Negre;Anne-Virginie Eggimann;Yves Beuzard;Jean-Antoine Ribeil

  • High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells

    Suzan Imren;Mary E. Fabry;Karen A. Westerman;Robert Pawliuk

  • Erosion Of The Chronic Myeloid Leukemia Stem Cell Pool By PPARγ Agonists

    Francis Relouzat;Marc Spenchian;Gérald Massonnet;Jean-Paul Beressi

Frequent Co-Authors

Noriaki Tanaka
Noriaki Tanaka Okayama University
Yves Beuzard
Yves Beuzard University of Paris-Saclay
Toshiyoshi Fujiwara
Toshiyoshi Fujiwara Okayama University
John E.J. Rasko
John E.J. Rasko Centenary Institute of Cancer Medicine and Cell Biology
Marina Cavazzana-Calvo
Marina Cavazzana-Calvo Necker-Enfants Malades Hospital
Connie J. Eaves
Connie J. Eaves University of British Columbia
Christof von Kalle
Christof von Kalle Charité - University Medicine Berlin
Masakiyo Sakaguchi
Masakiyo Sakaguchi Okayama University
Alain Fischer
Alain Fischer Collège de France
Suthat Fucharoen
Suthat Fucharoen Mahidol University

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