ArticleMolecular therapy. Methods & clinical development2019
PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector.
Article in Molecular therapy. Methods & clinical development, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers.
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Who cites it
37 citing papers in PubMed, 47 citations in OpenAlex.
- Clinical outcomes of lentiviral vector gene therapy for sickle cell disease.Blood advances · 2026Trial
- Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.Nature communications · 2025Trial
- Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.Blood advances · 2026Article
- Engineering single-vector logic-gated CAR T cells with transgene sizes beyond current limitations.Journal for immunotherapy of cancer · 2026Article
- CRISPR-MI and scRNA-Seq Reveal TREM2's Function in Monocyte Infiltration and Macrophage Apoptosis During Abdominal Aortic Aneurysm Development.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Article
- Quantification of intrinsic regulatory factors refines human hematopoietic progenitor definitions and reveals early erythroid lineage priming.Cell reports · 2025Article
- UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease.Blood advances · 2024Article
- Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.Biomedicines · 2024Article
- Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.Cells · 2024Review
- Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?Molecular therapy. Methods & clinical development · 2024Article
- CAR-T cell therapy targeting surface expression of TYRP1 to treat cutaneous and rare melanoma subtypes.Nature communications · 2024Article
- Prostaglandin E2 as transduction enhancer affects competitive engraftment of human hematopoietic stem and progenitor cells.Molecular therapy. Methods & clinical development · 2023Article
- Identification of a small molecule for enhancing lentiviral transduction of T cells.Molecular therapy. Methods & clinical development · 2023Article
- Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of Leukocyte Adhesion Deficiency Type 1.Human gene therapy · 2022Article
- Improving Lentiviral Transduction of Human Adipose-Derived Mesenchymal Stem Cells.Human gene therapy · 2022Article
- Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.Molecular therapy. Methods & clinical development · 2022Article
- Evolution of Gene Therapy, Historical Perspective.Hematology/oncology clinics of North America · 2022Review
- Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome.Frontiers in immunology · 2022Review
- Review
Corrections and comments
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Lentiviral vector (LV)-based hematopoietic stem and progenitor cell (HSPC) gene therapy is becoming a promising alternative to allogeneic stem cell transplantation for curing genetic diseases. Clinical trials are currently underway to treat sickle cell disease using LVs expressing designed anti-sickling globin genes. However, because of the large size and complexity of the human β-globin gene, LV products often have low titers and transduction efficiency, requiring large amounts to treat a single patient. Furthermore, transduction of patient HSPCs often fails to achieve a sufficiently high vector copy number (VCN) and transgene expression for clinical benefit. We therefore investigated the combination of two compounds (PGE2 and poloxamer synperonic F108) to enhance transduction of HSPCs with a clinical-scale preparation of Lenti/G-AS3-FB. Here, we found that transduction enhancers increased the
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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.