ArticleBlood2014
Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation.
Article in Blood, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01639690 (A Phase I Clinical Trial for the Treatment of ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene), which is not on this map. Cited by 32 papers.
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 I Clinical Trial for the Treatment of ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene
Who cites it
32 citing papers in PubMed, 63 citations in OpenAlex.
- The Functional Effect of Repeated Cryopreservation on Transduced CD34Human gene therapy methods · 2018Trial
- Metal Ion Signaling in Biomedicine.Chemical reviews · 2025Review
- An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA.Blood · 2025Article
- The transformative potential of HSC gene therapy as a genetic medicine.Gene therapy · 2023Review
- 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia.HemaSphere · 2022Article
- Globin vector regulatory elements are active in early hematopoietic progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Advancing the care of β-thalassaemia patients with novel therapies.Blood transfusion = Trasfusione del sangue · 2022Review
- Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.Nature medicine · 2022Article
- Review
- Iron metabolism: pathophysiology and pharmacology.Trends in pharmacological sciences · 2021Review
- Hematopoietic Stem Cells and Their Roles in Tissue Regeneration.International journal of stem cells · 2020Review
- Adenovirus vectors in hematopoietic stem cell genome editing.FEBS letters · 2019Review
- Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders.Nature communications · 2019Article
- Modeling blood diseases with human induced pluripotent stem cells.Disease models & mechanisms · 2019Review
- A Cetuximab-Mediated Suicide System in Chimeric Antigen Receptor-Modified Hematopoietic Stem Cells for Cancer Therapy.Human gene therapy · 2019Article
- Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches.Mediterranean journal of hematology and infectious diseases · 2019Review
- Gene Therapy For Beta-Thalassemia: Updated Perspectives.The application of clinical genetics · 2019Article
- β-Thalassemia intermedia: a comprehensive overview and novel approaches.International journal of hematology · 2018Review
- Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.Molecular therapy. Methods & clinical development · 2017Review
- Gene Therapy for β-Hemoglobinopathies.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 3 institutions in 2 countries.
Funding
Abstract
We conducted a pilot trial to investigate the safety and effectiveness of mobilizing CD34(+) hematopoietic progenitor cells (HPCs) in adults with β-thalassemia major. We further assessed whether thalassemia patient CD34(+) HPCs could be transduced with a globin lentiviral vector under clinical conditions at levels sufficient for therapeutic implementation. All patients tolerated granulocyte colony-stimulating factor well with minimal side effects. All cell collections exceeded 8 × 10(6) CD34(+) cells/kg. Using clinical grade TNS9.3.55 vector, we demonstrated globin gene transfer averaging 0.53 in 3 validation runs performed under current good manufacturing practice conditions. Normalized to vector copy, the vector-encoded β-chain was expressed at a level approximating normal hemizygous protein output. Importantly, stable vector copy number (0.2-0.6) and undiminished vector expression were obtained in NSG mice 6 months posttransplant. Thus, we validated a safe and effective procedure for β-globin gene transfer in thalassemia patient CD34(+) HPCs, which we will implement in the first US trial in patients with severe inherited globin disorders. This trial is registered at www.clinicaltrials.gov as #NCT01639690.
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Identifiers
What OpenQuestion holds
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 OpenQuestion graph.