Trial reportNature medicine2019
Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Registry-linked trialAbstract readClinical Trial, Phase IClinical Trial, Phase II
In one paragraph
Trial report in Nature medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03157804 (Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A), which is not on this map. Cited by 115 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
115citing papers in PubMed, 2 pooled it
–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.
Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)
Who cites it
115 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders.Nature communications · 2022Pooled it
- Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization.Annals of hematology · 2020Pooled it
- Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.Nature communications · 2023Trial
- HLA-haploidentical TCRαβ+/CD19+-depleted stem cell transplantation in children and young adults with Fanconi anemia.Blood advances · 2021Trial
- The potential of gene therapy to rescue telomeric and extratelomeric defects in X-linked dyskeratosis congenita.Molecular therapy. Advances · 2026Article
- Antimetabolites synergize with non-genotoxic antibody drug conjugate conditioning in hematopoietic stem cell lentiviral gene therapy.Molecular therapy. Advances · 2026Article
- Fanconi Anemia as a Window into Premalignant Field Cancerization of the Oral Mucosa.medRxiv : the preprint server for health sciences · 2026Article
- Molecular and therapeutic frontiers in anemia therapy.The Journal of clinical investigation · 2026Review
- Fanconi Anemia: Interplay Between DNA Repair Defects, Mitochondrial Dysfunction, and Oxidative Stress.Cells · 2026Review
- Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells.Cell stem cell · 2026Article
- Microglia replacement by peripheral delivery of CSF1R inhibitor-resistant hematopoietic cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Hematopoietic (stem) cells-The elixir of life?FEBS letters · 2026Review
- Adaptation of lentiviral vectors for viral gene therapy and their impact on host cell biology.Journal of translational medicine · 2026Review
- Development of evolutionarily conserved viral integration sites as safe harbors for human gene therapy.iScience · 2025Article
- Lentiviral-mediated panErbB CAR-T cell therapy against head and neck squamous cell carcinomas for patients with Fanconi anemia.Molecular therapy. Oncology · 2025Article
- Tracking Cytopenias inmedRxiv : the preprint server for health sciences · 2025Article
- CRISPR-based therapeutic genome editing for inherited blood disorders.Nature reviews. Drug discovery · 2025Review
- A rescue fanconi anemia humanized mouse model with endogenous FA mutation and high human hematopoietic stem cell chimerism.Molecular therapy. Methods & clinical development · 2025Article
- Epithelial competition determines gene therapy potential to suppress Fanconi Anemia oral cancer risk.PLoS computational biology · 2025Article
- Epithelial competition determines gene therapy potential to suppress Fanconi Anemia oral cancer risk.bioRxiv : the preprint server for biology · 2025Article
55 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
37 authors.
Paula RíoHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Susana NavarroHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Wei WangDivision of Translational Oncology, German Cancer Research Center and National Center for Tumor Diseases, Heidelberg, Germany.
Rebeca Sánchez-DomínguezHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Roser M PujolCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
José C SegoviaHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Massimo BoglioloCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
Eva MerinoCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
Ning WuDivision of Translational Oncology, German Cancer Research Center and National Center for Tumor Diseases, Heidelberg, Germany.
Rocío SalgadoServicio de Hematología, Hospital Universitario Fundación Jiménez Diaz, Instituto de Investigaciones Sanitarias de la Fundación Jiménez Díaz, Madrid, Spain.
María L LamanaHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Rosa M YañezHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
José A CasadoHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Yari GiménezHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Francisco J Román-RodríguezHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Lara ÁlvarezHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Omaira AlberquillaHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Anna RaimbaultUniversité de Paris (IRSL, INSERM, CNRS), Paris, France.
Guillermo GuenecheaHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
M Luz LozanoHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Laura CerratoHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Miriam HernandoHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Eva GálvezCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
Raquel HladunServicio de Oncología y Hematología Pediátricas, Hospital Universitari Vall d'Hebron, Barcelona, Spain.
Irina GiraltVall d'Hebron Institut de Recerca, Barcelona, Spain.
Jordi BarquineroVall d'Hebron Institut de Recerca, Barcelona, Spain.
Anne GalyGénéthon, Evry, France.
Nagore García de AndoínHospital Universitario Donostia, San Sebastián, Spain.
Ricardo LópezOsakidetza Basque Health Service, Pediatric Oncology and Hematology Unit, Cruces University Hospital, Barakaldo, Spain.
Albert CataláCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
Jonathan D SchwartzRocket Pharma, New York, NY, USA.
Jordi SurrallésCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
Jean SoulierUniversité de Paris (IRSL, INSERM, CNRS), Paris, France.
Manfred SchmidtDivision of Translational Oncology, German Cancer Research Center and National Center for Tumor Diseases, Heidelberg, Germany.
Cristina Díaz de HerediaServicio de Oncología y Hematología Pediátricas, Hospital Universitari Vall d'Hebron, Barcelona, Spain.
Julián SevillaCentro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.ORCID http://orcid.org/0000-0002-6852-1860
Juan A BuerenHematopoietic Innovative Therapies Division, Centro de investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain. juan.bueren@ciemat.es.ORCID http://orcid.org/0000-0002-3228-7013
Funding
SUBTHRESHOLD MUSCLE AFFERENT INPUT TO DORSAL HORNF06TW002012 · FIC · UNIVERSITY OF CALIFORNIA DAVIS · PI KAUFMAN, MARC PETER · 1995 to 1995
–Abstract
Fanconi anemia (FA) is a DNA repair syndrome generated by mutations in any of the 22 FA genes discovered to date
Indexed as
Genetic TherapyHematopoietic Stem Cell TransplantationAdolescentAdultBone Marrow CellsChildChild, PreschoolFanconi AnemiaFanconi Anemia Complementation Group A ProteinFemaleGenetic VectorsHematopoietic Stem CellsHumansInfantLentivirusMaleFanconi Anemia Complementation Group A Protein
Identifiers
PMID
31501599What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
NCT03157804ClinicalTrials.gov
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 OpenQuestion graph.