ArticleMolecular therapy. Methods & clinical development2015
Amelioration of murine sickle cell disease by nonablative conditioning and γ-globin gene-corrected bone marrow cells.
Article in Molecular therapy. Methods & clinical development, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 18 citations in OpenAlex.
- Article
- In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.JCI insight · 2022Article
- Development and characterization of a preclinical total marrow irradiation conditioning-based bone marrow transplant model for sickle cell disease.Frontiers in oncology · 2022Article
- Correction of murine sickle cell disease by allogeneic haematopoietic cell transplantation with anti-3rd party veto cells.Bone marrow transplantation · 2021Article
- Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.Nature communications · 2021Article
- Enabling allogeneic therapies: CIRM-funded strategies for immune tolerance and immune evasion.Stem cells translational medicine · 2020Review
- The mRNA-Binding Protein IGF2BP1 Restores Fetal Hemoglobin in Cultured Erythroid Cells from Patients with β-Hemoglobin Disorders.Molecular therapy. Methods & clinical development · 2020Article
- High level of fetal-globin reactivation by designed transcriptional activator-like effector.Blood advances · 2020Article
- Low-Dose Busulfan Reduces Human CD34Molecular therapy. Methods & clinical development · 2019Article
- Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies.Molecular therapy. Methods & clinical development · 2018Article
- A clinically meaningful fetal hemoglobin threshold for children with sickle cell anemia during hydroxyurea therapy.American journal of hematology · 2017Article
- Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- A Child as a Donor for Hematopoietic Stem Cell Transplantation: Bioethical Justification-A Case Study on Sickle Cell Disease.Case reports in hematology · 2017Article
- Precipitating factors and targeted therapies in combating the perils of sickle cell disease--- A special nutritional consideration.Nutrition & metabolism · 2016Review
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 1 country.
Funding
Abstract
Patients with severe sickle cell disease (SCD) are candidates for gene therapy using autologous hematopoietic stem cells (HSCs), but concomitant multi-organ disease may contraindicate pretransplant conditioning with full myeloablation. We tested whether nonmyeloablative conditioning, a regimen used successfully for allogeneic bone marrow transplantation of adult SCD patients, allows engraftment of γ-globin gene-corrected cells to a therapeutic level in the Berkeley mouse model of SCD. Animals transplanted according to this regimen averaged 35% engraftment of transduced hematopoietic stem cells with an average vector copy < 2.0. Fetal hemoglobin (HbF) levels ranged from 20 to 44% of total hemoglobin and approximately two-thirds of circulating red blood cells expressed HbF detected by immunofluorescence (F-cells). Gene therapy treatment of SCD mice ameliorated anemia, reduced hyperleukocytosis, improved renal function, and reduced iron accumulation in liver, spleen, and kidneys. Thus, modest levels of chimerism with donor cells expressing high levels of HbF from an insulated γ-globin lentiviral vector can improve the pathology of SCD in mice, thereby illustrating a potentially safe and effective strategy for gene therapy in humans.
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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.