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Blood|December 9, 2004
Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trialManfred Schmidt, Salima Hacein-Bey-Abina, Manuela Wissler, et al.
The Journal of Experimental Medicine|March 14, 2023
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiencyRomane Thouenon, Loïc Chentout, Nidia Moreno-Corona, et al.
Blood|September 22, 2005
Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicityChantal Lagresle-Peyrou, Frank Yates, Michèle Malassis-Séris, et al.
Blood Advances|February 14, 2019
Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiencyOrnellie Bernadin, Fouzia Amirache, Anais Girard-Gagnepain, et al.
Blood|February 5, 2021
A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndromeMarianne Delville, Florence Bellier, Juliette Leon, et al.
Biomedicines|July 27, 2024
Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem CellsNataël Sorel, Francisco Díaz-Pascual, Boris Bessot, et al.
The Journal of Allergy and Clinical Immunology|July 30, 2015
An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, and MPN domains 1 (MYSM1) in human hematopoiesis and lymphocyte differentiationTangui Le Guen, Fabien Touzot, Isabelle André-Schmutz, et al.
Cellular & Molecular Immunology|June 12, 2021
A DL-4- and TNFα-based culture system to generate high numbers of nonmodified or genetically modified immunotherapeutic human T-lymphoid progenitorsRanjita Devi Moirangthem, Kuiying Ma, Sabrina Lizot, et al.
Haematologica|February 24, 2018
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusionChantal Lagresle-Peyrou, François Lefrère, Elisa Magrin, et al.
The Journal of Allergy and Clinical Immunology|December 17, 2013
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndromeNiek P van Til, Roya Sarwari, Trudi P Visser, et al.
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