ArticleNature medicine2024
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Open access · greenAbstract read
In one paragraph
Article in Nature medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 1 pooled it
13.8field-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
16 citing papers in PubMed, 1 synthesis or guideline pooled it, 25 citations in OpenAlex.
- Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations.Bone marrow transplantation · 2025Guideline
- Clinical, immunological, and molecular characteristics of severe combined immune deficiency in China.Human genetics · 2026Article
- AAV-based gene therapies for neovascular AMD.Gene therapy · 2026Review
- Addressing clinical uncertainties in ATMP reimbursement: a review of methodological guidelines and European practice.Frontiers in pharmacology · 2026Review
- Characterizing Gene Therapy Medicinal Products Post-Marketing Safety Reports Based on WHO Pharmacovigilance Database, VigiBase.Therapeutic innovation & regulatory science · 2025Article
- Characterizing Gene Therapy Medicinal Products Post-Marketing Safety Reports Based on WHO Pharmacovigilance Database, VigiBase.Therapeutic innovation & regulatory science · 2025Article
- Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- The Era of Gene Therapy: The Advancement of Lentiviral Vectors and Their Pseudotyping.Viruses · 2025Review
- Current landscape of vector safety and genotoxicity after hematopoietic stem or immune cell gene therapy.Leukemia · 2025Review
- Neutralizing Antibodies: Role in Immune Response and Viral Vector Based Gene Therapy.International journal of molecular sciences · 2025Review
- Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Inborn Errors of Immunity Presenting with Early-Onset Severe Atopy.Medicina (Kaunas, Lithuania) · 2025Review
- Entering the playing field: Therapy for multiple sulfatase deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Omenn Syndrome can Occur during Enzyme Therapy for Adenosine Deaminase Deficiency.Journal of clinical immunology · 2024Article
- A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.Nature communications · 2024Article
- Unlocking the full potential of rare disease drug development: exploring the not-for-profit sector's contributions to drug development and access.Frontiers in pharmacology · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
54 authors at 19 institutions in 8 countries.
Maddalena MigliavaccaSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Federica BarzaghiSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Claudia FossatiSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paola M V RancoitaUniversity Centre for Statistics in the Biomedical Sciences (CUSSB), Vita-Salute San Raffaele University, Milan, Italy.
Michela GabaldoFondazione Telethon, Milan, Italy.
Francesca DionisioSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Stefania GiannelliSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Federica Andrea SalerioSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Francesca FerruaSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Francesca TucciSan 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.ORCID 0000-0003-3514-666X
Vera GalloSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Salvatore RecuperoSan 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.
Roberta PajnoSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Maria SambucoSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Alessio PrioloSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0001-9394-7292
Chiara FerriUniversità Vita-Salute San Raffaele, Milan, Italy.
Vittoria GarellaUniversità Vita-Salute San Raffaele, Milan, Italy.
Ilaria MontiSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paolo SilvaniDepartment of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Silvia DarinSan 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.
Ambra CortiSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Stefano ZancanFondazione Telethon, Milan, Italy.
Margherita LeviFondazione Telethon, Milan, Italy.
Daniela CesanaSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0001-6366-4224
Filippo CarlucciDepartment of Medical Biotechnologies, University of Siena, Siena, Italy.
Anna Pituch-NoworolskaDepartment of Immunology, University Children's Hospital of Cracow, Cracow, Poland.
Dalia AbdElazizDepartment of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt.ORCID 0000-0002-7830-7008
Ulrich BaumannDepartment of Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany.ORCID 0000-0002-0162-0266
Andrea FinocchiResearch Unit of Primary Immunodeficiencies, Academic Department of Pediatrics, Bambino Gesù Children's Hospital, Scientific Institute for Research and Healthcare (IRCCS), Rome, Italy.
Caterina CancriniResearch Unit of Primary Immunodeficiencies, Academic Department of Pediatrics, Bambino Gesù Children's Hospital, Scientific Institute for Research and Healthcare (IRCCS), Rome, Italy.
Saverio LadoganaPaediatric Onco-haematology Unit, 'Casa Sollievo della Sofferenza' Hospital, IRCCS, San Giovanni Rotondo, Italy.
Andrea MeinhardtDepartment of Pediatric Hematology and Oncology, Medical Center, University Hospital Giessen, Giessen, Germany.ORCID 0000-0002-7464-0751
Isabelle MeytsLaboratory of Inborn Errors of Immunity, Department of Microbiology, Immunology and Transplantation, KU Leuven, Childhood Immunology, Department of Pediatrics, UZ Leuven, Leuven, Belgium.ORCID 0000-0003-1214-0302
Davide MontinDepartment of Pediatric and Public Health Sciences, University of Torino, Turin, Italy.
Lucia Dora NotarangeloMedical Direction, Children's Hospital, ASST-Spedali Civili, Brescia, Italy.
Fulvio PortaPediatric Oncology-Hematology and BMT Unit, Spedali Civili di Brescia, Brescia, Italy.
Marlène PasquetPediatric Hematology and Immunology, Children's Hospital, Toulouse, France.
Carsten SpeckmannInstitute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Faculty of Medicine, Medical Center, University of Freiburg, Freiburg, Germany.
Polina StepenskyDepartment of Bone Marrow Transplantation and Cancer Immunotherapy and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Alberto TommasiniDepartment of Pediatrics, Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.
Marco RabusinDepartment of Pediatrics, Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.
Zeynep KarakasDepartment of Pediatrics, Hematology/Oncology Unit, Istanbul School of Medicine, Istanbul University, Istanbul, Turkey.
Miguel GalicchioAllergy and Immnunology Service, Hospital de Niños VJ Vilela, Rosario, Argentina.
Lucia LeonardiDepartment of Maternal, Infantile and Urological Sciences, Sapienza University of Rome, Rome, Italy.
Marzia DuseDepartment of Maternal, Infantile and Urological Sciences, Sapienza University of Rome, Rome, Italy.ORCID 0000-0001-9958-2570
Sukru Nail GunerDivision of Pediatric Allergy and Immunology, Faculty of Medicine, Necmettin Erbakan University, Konya, Turkey.ORCID 0000-0002-8860-6132
Clelia Di SerioUniversity Centre for Statistics in the Biomedical Sciences (CUSSB), Vita-Salute San Raffaele University, Milan, Italy.
Fabio CiceriSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0003-0873-0123
Maria Ester BernardoSan 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. aiuti.alessandro@hsr.it.ORCID 0000-0002-5398-1717
Maria Pia CicaleseSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Vita-Salute San Raffaele University · ITThe San Raffaele Telethon Institute for Gene Therapy · ITTelethon Foundation · ITAzienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia · ITIRCCS Materno Infantile Burlo Garofolo · ITSapienza University of Rome · ITUniversity of Rome Tor Vergata · ITCairo University · EGCasa Sollievo della Sofferenza · ITCracow University of Technology · PLHadassah Medical Center · ILIstanbul University · TRMedizinische Hochschule Hannover · DENecmettin Erbakan University · TRUniversitair Ziekenhuis Leuven · BEUniversitätsklinikum Gießen und Marburg · DEUniversity of Freiburg · DEUniversity of Siena · ITUniversity of Turin · IT
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34
Indexed as
AgammaglobulinemiaHematopoietic Stem Cell TransplantationSevere Combined ImmunodeficiencyAdenosine DeaminaseBusulfanGenetic TherapyHumansRetroviridaeAdenosine DeaminaseBusulfan
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