ArticleNature medicine2022
Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.
Open access · greenAbstract readClinical Trial, Phase I
In one paragraph
Article in Nature medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
0numbers the graph read from it
0cells of the map it votes in
36citing papers in PubMed
8.4field-weighted citation impact, top 2% 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
36 citing papers in PubMed, 47 citations in OpenAlex.
- 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
- Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.Molecular therapy. Advances · 2026Review
- Development of evolutionarily conserved viral integration sites as safe harbors for human gene therapy.iScience · 2025Article
- 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
- Recent advances in mucopolysaccharidosis IVA treatment.Orphanet journal of rare diseases · 2025Review
- Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Assessment of different promoters in lentiviral vectors for expression of the N-acetyl-galactosamine-6-sulfate sulfatase gene.Journal of human genetics · 2025Article
- Gene therapy for sickle cell disease: recent advances, clinical trials and future directions.Cytotherapy · 2025Review
- Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models.Molecular therapy. Methods & clinical development · 2025Article
- Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overview.Regenerative therapy · 2025Review
- Preclinical efficacy and safety evaluation of BD211 autologous CD34Frontiers in cell and developmental biology · 2025Article
- Cardiac injury caused by iron overload in thalassemia.Frontiers in pediatrics · 2025Review
- Review
- Long-term tracking of haematopoietic clonal dynamics and mutations in non-human primate undergoing transplantation of lentivirally barcoded haematopoietic stem and progenitor cells.British journal of haematology · 2024Article
- Lentiviral Vector-MediatedHuman gene therapy · 2024Article
- Article
- CRISPR technology in human diseases.MedComm · 2024Review
- Viral-mediated gene therapy in pediatric neurological disorders.World journal of pediatrics : WJP · 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
32 authors at 7 institutions in 3 countries.
Farid BouladStem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Aurelio MaggioDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Xiuyan WangCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Paolo MoiOspedale Pediatrico Microcitemie 'A.Cao', A.O. 'G.Brotzu', Cagliari, Italy.
Santina AcutoDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Friederike KogelCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID http://orcid.org/0000-0002-8888-2268
Chayamon TakpraditStem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Susan ProckopStem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Jorge Mansilla-SotoCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Annalisa CabrioluCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID http://orcid.org/0000-0002-8216-2120
Ashlesha OdakCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID http://orcid.org/0000-0001-5595-0394
Jinrong QuCell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Keyur ThummarCell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Fang DuCell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Lingbo ShenCell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Simona RasoDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Rita BaroneDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Rosario Di MaggioDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Lorella PitroloDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Antonino GiambonaDepartment of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
Maura MingoiaOspedale Pediatrico Microcitemie 'A.Cao', A.O. 'G.Brotzu', Cagliari, Italy.
John K EverettDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Pascha HokamaDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Aoife M RocheDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Vito Adrian CantuDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0002-3168-5140
Hriju AdhikariDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.ORCID http://orcid.org/0000-0001-9959-298X
Shantan ReddyDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Eric BouhassiraDepartment of Cell Biology, Albert Einstein College of Medicine, Bronx, New York, NY, USA.
Narla MohandasLaboratory of Red Cell Physiology, New York Blood Center, New York, NY, USA.
Frederic D BushmanDepartment of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Isabelle RivièreCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Michel SadelainCenter for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA. m-sadelain@ski.mskcc.org.ORCID http://orcid.org/0000-0002-9031-8025
Memorial Sloan Kettering Cancer Center · USOspedale Vincenzo Cervello · ITUniversity of Pennsylvania · USOspedale Microcitemico · ITAlbert Einstein College of Medicine · USNew York Blood Center · USSiriraj Hospital · TH
Funding
X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4MNCI NIH HHS P30 CA008748
Abstract
β-Thalassemias are inherited anemias that are caused by the absent or insufficient production of the β chain of hemoglobin. Here we report 6-8-year follow-up of four adult patients with transfusion-dependent β-thalassemia who were infused with autologous CD34
Indexed as
Genetic VectorsAdolescentAdultAntigens, CD34beta-ThalassemiaBlood TransfusionFemaleGenetic TherapyGlobinsHumansLentivirusMaleTransduction, GeneticTransplantation ConditioningYoung AdultAntigens, CD34Globins
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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.