ArticleMolecular therapy : the journal of the American Society of Gene Therapy2021
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 23 citations in OpenAlex.
- Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.Nature medicine · 2025Trial
- Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy for Sickle Cell Disease and Transfusion-Dependent Thalassemia.Clinics in laboratory medicine · 2026Review
- The strategies and advances of mRNA translation booster.Asian journal of pharmaceutical sciences · 2025Review
- Culturing Potential: advances in ex vivo cell culture systems for haematopoietic cell-based regenerative therapies.Regenerative therapy · 2025Review
- Gene therapy for sickle cell disease: recent advances, clinical trials and future directions.Cytotherapy · 2025Review
- Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations.Molecular therapy. Nucleic acids · 2025Article
- An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA.Blood · 2025Article
- Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Article
- Genetic alteration of SJ293TS cells and modification of serum-free media enhances lentiviral vector production.Molecular therapy. Methods & clinical development · 2024Article
- Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia byCells · 2023Article
- In vivo hematopoietic stem cell modification by mRNA delivery.Science (New York, N.Y.) · 2023Article
- Article
- Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Inclusion of a short hairpin RNA targetingHaematologica · 2021Article
- Old versus new gene therapy for globin disorders.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Current therapeutic landscape of β-thalassemia: focus on gene therapy.Therapeutic advances in rare diseaseReview
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Authors and funding
24 authors at 3 institutions in 2 countries.
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No grant is acknowledged in the PubMed record.
Abstract
Ongoing clinical trials for treatment of beta-globinopathies by gene therapy involve the transfer of the beta-globin gene, which requires integration of three to four copies per genome in most target cells. This high proviral load may increase genome toxicity, potentially limiting the safety of this therapy and relegating its use to total body myeloablation. We hypothesized that introducing an additional hypersensitive site from the locus control region, the complete sequence of the second intron of the beta-globin gene, and the ankyrin insulator may enhance beta-globin expression. We identified a construct, ALS20, that synthesized significantly higher adult hemoglobin levels than those of other constructs currently used in clinical trials. These findings were confirmed in erythroblastic cell lines and in primary cells isolated from sickle cell disease patients. Bone marrow transplantation studies in beta-thalassemia mice revealed that ALS20 was curative at less than one copy per genome. Injection of human CD34
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