Trial reportNature communications2025
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.
3 registry-linked trialsAbstract readClinical Trial, Phase IIClinical Trial, Phase I
In one paragraph
Trial report in Nature communications, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 12 papers.
0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients With Sickle Cell Disease (SCD)
NCT07432867 phase1 / phase2recruitingnot on this mapstarted 2026, after this paper: background citation
A Phase 1/2 Open Label Cohort Study Evaluating the Efficacy and Safety of Gene Therapy of the Sickle Cell Disease (SCD) by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains Autologous CD34+ Cells Transduced ex Vivo by the Bifunctional βAS3m/miR7m Lentiviral Vector Expressing the Therapeutical Beta-globin βAS3m and a Micro-RNA (miRNA) Targeting Specifically the Endogenous βS-globin mRNA.
Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients
Who cites it
12 citing papers in PubMed.
- The blood remembers: HSC-iM and the cancer continuum.Blood cancer journal · 2026Article
- Article
- The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care.Cells · 2026Review
- What we thought we knew about TNF-And what we now must re-learn.Stem cell reports · 2026Article
- Erythropoiesis in health and disease: Distinguishing defective and ineffective erythropoiesis.HemaSphere · 2026Review
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Circular single stranded DNA potentiates non-viral gene insertion in hematopoietic stem and progenitor cells.Nature communications · 2025Article
- Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy.Cureus · 2025Review
- Hematopoietic stem cell therapy with gene modification to treat sickle cell disease.Stem cells translational medicine · 2025Review
- Dissecting the epigenetic regulation of the fetal hemoglobin genes to unravel a novel therapeutic approach for β-hemoglobinopathies.Nucleic acids research · 2025Article
- Hypoxia, Inflammation, and Cytokine Crosstalk in Sickle Cell Disease: From Mechanisms to Modulation- A Narrative Review.Pediatric health, medicine and therapeutics · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
34 authors.
Steicy SobrinoLaboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.
Laure JosephDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Elisa MagrinDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Anne ChalumeauLaboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.ORCID http://orcid.org/0000-0003-3404-1401
Nicolas HebertUniv Paris Est Créteil, IMRB, Laboratory of Excellence LABEX GRex, Créteil, France.ORCID http://orcid.org/0000-0003-0232-831X
Alice CorsiaDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Adeline DenisLaboratory of Human Lymphohematopoiesis, INSERM, Imagine Institute, Paris, France.
Cécile RoudautDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Clotilde AusselDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Olivia LeblancDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Mégane BrussonLaboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.ORCID http://orcid.org/0000-0001-8567-444X
Tristan FelixLaboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.ORCID http://orcid.org/0000-0002-5331-3153
Jean-Sebastien DianaDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Angelina PetrichenkoDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0003-0600-0709
Jana El EtriLaboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.
Auria GodardAix Marseille Univ, CNRS, EFS, ADES, Labex GR-Ex, Marseille, France.
Eden TibiDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Sandra ManceauDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Jean Marc TreluyerCentre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Fulvio MavilioDepartment of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.ORCID http://orcid.org/0000-0003-0459-4320
Frederic D BushmanDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0003-4740-4056
Ambroise MarcaisDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.ORCID http://orcid.org/0000-0002-6844-7920
Martin CastellePediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France.
Benedicte NevenPediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France.
Olivier HermineDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Sylvain RenolleauDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.ORCID http://orcid.org/0000-0002-0638-5613
Alessandra MagnaniDepartement of Biotherapy Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Groupe Hospitalier Universitaire Centre, Université Paris Cité, Paris, France.
Vahid AsnafiLaboratory of Onco-Hematology, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.ORCID http://orcid.org/0000-0002-6255-5314
Wassim El NemerAix Marseille Univ, CNRS, EFS, ADES, Labex GR-Ex, Marseille, France.
Pablo BartolucciUniv Paris Est Créteil, IMRB, Laboratory of Excellence LABEX GRex, Créteil, France.
Emmanuelle SixLaboratory of Human Lymphohematopoiesis, INSERM, Imagine Institute, Paris, France.ORCID http://orcid.org/0000-0001-7806-0968
Michaela SemeraroCentre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Annarita MiccioLaboratory of Chromatin and Gene Regulation During Development, University Paris Cite, UMR1163 INSERM, Imagine Institute, Paris, France.ORCID http://orcid.org/0000-0002-3409-9665
Marina CavazzanaDepartement 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.ORCID http://orcid.org/0000-0002-0264-0891
Funding
AFM-Téléthon (French Muscular Dystrophy Association) 23879Agence Nationale de la Recherche (French National Research Agency) ANR-10-IAHU-01
Abstract
In sickle cell disease (SCD), the β6
Indexed as
Anemia, Sickle CellHematopoietic Stem CellsHematopoietic Stem Cell TransplantationInflammationAdolescentAdultbeta-GlobinsCell LineageChildFemaleGenetic TherapyGenetic VectorsHumansLentivirusMaleYoung Adultbeta-Globins
Identifiers
What Socratic holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.