ArticleMolecular therapy. Methods & clinical development2018
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia.
Article in Molecular therapy. Methods & clinical development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 23 citations in OpenAlex.
- Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.The Lancet. Haematology · 2019Trial
- Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.Nature medicine · 2019Trial
- Capsid stabilization reprograms the nuclear fate of the HIV genome.Science advances · 2026Article
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Prostaglandin E2 as transduction enhancer affects competitive engraftment of human hematopoietic stem and progenitor cells.Molecular therapy. Methods & clinical development · 2023Article
- Gene Therapy and Gene Editing for β-Thalassemia.Hematology/oncology clinics of North America · 2023Review
- Autologous, lentivirus-modified, T-rapa cell "micropharmacies" for lysosomal storage disorders.EMBO molecular medicine · 2022Article
- Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review).Biomedical reports · 2020Review
- Gene Therapy of the Hemoglobinopathies.HemaSphere · 2020Review
- Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.Molecular therapy. Methods & clinical development · 2020Article
- Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.Blood advances · 2020Article
- Episomal vectors based on S/MAR and the β-globin Replicator, encoding a synthetic transcriptional activator, mediate efficient γ-globin activation in haematopoietic cells.Scientific reports · 2019Article
- Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges.Molecular diagnosis & therapy · 2019Review
- Cardiomyocyte Progenitor Cells as a Functional Gene Delivery Vehicle for Long-Term Biological Pacing.Molecules (Basel, Switzerland) · 2019Article
- Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches.Mediterranean journal of hematology and infectious diseases · 2019Review
- Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives.Frontiers in pediatrics · 2019Review
- Gene Therapy for Hemoglobinopathies.Human gene therapy · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
18 authors at 8 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy clinical trials require rigorous non-clinical studies in the most relevant models to assess the benefit-to-risk ratio. To support the clinical development of gene therapy for β-thalassemia, we performed
Indexed as
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.