Trial reportNature medicine2019
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Abstract 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. Cited by 140 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
140citing papers in PubMed, 2 pooled it
21.4field-weighted citation impact, top 1% of its field
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
140 citing papers in PubMed, 2 syntheses or guidelines pooled it, 224 citations in OpenAlex.
- Risk of cancer in patients with thalassemia and sickle cell disease: a systematic review.Annals of medicine · 2025Pooled it
- A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders.Nature communications · 2022Pooled it
- Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.Nature communications · 2025Trial
- Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.Nature communications · 2023Trial
- Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.The New England journal of medicine · 2021Trial
- Sleeping Beauty-engineered CAR T cells achieve antileukemic activity without severe toxicities.The Journal of clinical investigation · 2020Trial
- Update of the decision-making algorithm on selecting transfusion-dependent β-thalassemic patients for gene therapy approaches: joint consensus report on behalf of EHA-specialized working group and EBMT hemoglobinopathies working party.Bone marrow transplantation · 2026Article
- Thalassaemia.Nature reviews. Disease primers · 2026Review
- New Gene Therapy Strategy for β-Thalassemia.Stem cell reviews and reports · 2026Review
- Disease Burden in Transfusion-Dependent β-Thalassemia (TDT) Patients and Caregivers in Italy: Results of a Cross-Sectional Descriptive Online Survey.Drugs - real world outcomes · 2026Article
- Review
- Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells.Cell stem cell · 2026Article
- Hematopoietic (stem) cells-The elixir of life?FEBS letters · 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
- Genetic Engineering in Hematopoietic Stem Cells for β-Hemoglobinopathies Treatment: Advances, Challenges, and Clinical Translation.International journal of hematology-oncology and stem cell research · 2025Review
- Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overview.Regenerative therapy · 2025Review
- Current research status of third-generation sequencing technology in thalassemia detection.Frontiers in pediatrics · 2025Review
- Cardiac injury caused by iron overload in thalassemia.Frontiers in pediatrics · 2025Review
- Article
80 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
38 authors at 9 institutions in 2 countries.
Sarah MarktelSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Samantha ScaramuzzaSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maria Pia CicaleseSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Fabio GiglioHaematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Stefania GalimbertiCenter of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milan-Bicocca, Milan, Italy.
Maria Rosa LidonniciSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Valeria CalbiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Andrea AssanelliHaematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maria Ester BernardoSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Claudia RossiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Andrea CalabriaSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID http://orcid.org/0000-0003-3515-3384
Raffaella MilaniBlood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Salvatore GattilloBlood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Fabrizio BenedicentiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Giulio SpinozziSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Annamaria AprileSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Alessandra BergamiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Miriam CasiraghiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Giulia ConsiglieriSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Nicoletta MaseraPediatric Department University of Milano-Bicocca, San Gerardo Hospital, Monza, Italy.
Emanuela D'AngeloPediatric Clinic/DH, Fondazione IRCCS Ca' Granda, Milan, Italy.
Nadia MirraPediatric Clinic/DH, Fondazione IRCCS Ca' Granda, Milan, Italy.
Raffaella OrigaOspedale Pediatrico Microcitemico "A.Cao", A.O. "G.Brotzu", Cagliari, Italy.
Immacolata TartaglioneDepartment of Woman, Child and General and Specialist Surgery, Università degli studi della Campania "Luigi Vanvitelli", Napoli, Italy.
Silverio PerrottaDepartment of Woman, Child and General and Specialist Surgery, Università degli studi della Campania "Luigi Vanvitelli", Napoli, Italy.
Robert WinterDepartment of Chemical Pathology, Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Milena CoppolaBlood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Gianluca ViarengoImmunohematology and Transfusion Medicine Service, Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy.
Luca SantoleriBlood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Giovanna GraziadeiRare Disease Center, Fondazione IRCCS Ca' Granda, University of Milan, Milan, Italy.
Michela GabaldoSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maria Grazia ValsecchiCenter of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milan-Bicocca, Milan, Italy.
Eugenio MontiniSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Luigi NaldiniSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maria Domenica CappelliniRare Disease Center, Fondazione IRCCS Ca' Granda, University of Milan, Milan, Italy.
Fabio CiceriSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Alessandro AiutiSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Giuliana FerrariSan Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. ferrari.giuliana@hsr.it.ORCID http://orcid.org/0000-0003-0790-3133
Vita-Salute San Raffaele University · ITThe San Raffaele Telethon Institute for Gene Therapy · ITUniversity of Milano-Bicocca · ITFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · ITUniversity of Campania "Luigi Vanvitelli" · ITUniversity of Milan · ITGreat Ormond Street Hospital for Children NHS Foundation Trust · GBOspedale Microcitemico · ITPoliclinico San Matteo Fondazione · IT
Funding
No grant is acknowledged in the PubMed record.
Abstract
ß-thalassemia is caused by ß-globin gene mutations resulting in reduced (β
Indexed as
Blood TransfusionGenetic TherapyHematopoietic Stem Cell TransplantationAdolescentAdultbeta-ThalassemiaBone and BonesChildChild, PreschoolFemaleHematopoietic Stem CellsHumansMaleTreatment Outcome
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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.