Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.

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Title: Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.
Authors: Sobrino S; Laboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.; Laboratory of Human Lymphohematopoiesis, INSERM, Imagine Institute, Paris, France., Joseph L; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France., Magrin E; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France., Chalumeau A; Laboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France., Hebert N; Univ Paris Est Créteil, IMRB, Laboratory of Excellence LABEX GRex, Créteil, France.; Etablissement Français du Sang, Créteil, France., Corsia A; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Department of Adult Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Laboratory of Excellence LABEX GRex, Paris, France., Denis A; Laboratory of Human Lymphohematopoiesis, INSERM, Imagine Institute, Paris, France., Roudaut C; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France., Aussel C; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France., Leblanc O; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France., Brusson M; Laboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France., Felix T; Laboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France., Diana JS; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France., Petrichenko A; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., El Etri J; Laboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.; Laboratory of Human Lymphohematopoiesis, INSERM, Imagine Institute, Paris, France., Godard A; Aix Marseille Univ, CNRS, EFS, ADES, Labex GR-Ex, Marseille, France., Tibi E; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France., Manceau S; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Department of Adult Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Laboratory of Excellence LABEX GRex, Paris, France., Treluyer JM; Centre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.; Université Paris Cité, Paris, France., Mavilio F; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy., Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Marcais A; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Department of Adult Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Laboratory of Excellence LABEX GRex, Paris, France., Castelle M; Pediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France., Neven B; Pediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France., Hermine O; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Department of Adult Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Laboratory of Excellence LABEX GRex, Paris, France., Renolleau S; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Department of Adult Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Laboratory of Excellence LABEX GRex, Paris, France., Magnani A; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France., Asnafi V; Laboratory of Onco-Hematology, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France., El Nemer W; Aix Marseille Univ, CNRS, EFS, ADES, Labex GR-Ex, Marseille, France., Bartolucci P; Univ Paris Est Créteil, IMRB, Laboratory of Excellence LABEX GRex, Créteil, France.; Paris-East Créteil University, Henri Mondor University Hospitals, APHP, Sickle Cell Referral Center-UMGGR, Créteil, France., Six E; Laboratory of Human Lymphohematopoiesis, INSERM, Imagine Institute, Paris, France., Semeraro M; Centre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.; Université Paris Cité, Inserm, Pharmacologie et évaluations des thérapeutiques chez l'enfant et la femme enceinte, Paris, France., Miccio A; Laboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France., Cavazzana M; Departement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France. m.cavazzana@aphp.fr.; Biotherapy Clinical Investigation Center, AP-HP, INSERM, Institut Imagine, Paris, France. m.cavazzana@aphp.fr.
Source: Nature communications [Nat Commun] 2025 Apr 01; Vol. 16 (1), pp. 3137. Date of Electronic Publication: 2025 Apr 01.
Publication Type: Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I
Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:2041-1723
DOI:10.1038/s41467-025-58321-4