ArticleBlood advances2021
Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice.
Article in Blood advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed, 20 citations in OpenAlex.
- Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease.Cell stem cell · 2026Article
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- De novo design of miniproteins targeting GPCRs.Nature · 2026Article
- In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery.Nature biomedical engineering · 2026Article
- GroβT rapidly and reliably mobilizes primitive hematopoietic stem and progenitor cells in nonhuman primates.Molecular therapy. Methods & clinical development · 2025Article
- Article
- Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors.Molecular therapy. Nucleic acids · 2024Article
- Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Auto-expansion ofMolecular therapy. Methods & clinical development · 2024Article
- A simplified G-CSF-free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model.Blood advances · 2024Article
- Targeting CXCR4, VLA-4, and CXCR2 for hematopoietic stem cell mobilization.Blood advances · 2024Article
- Homing and Engraftment of Hematopoietic Stem Cells Following Transplantation: A Pre-Clinical Perspective.Current oncology (Toronto, Ont.) · 2024Review
- Article
- In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.JCI insight · 2022Article
- Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells.Cell · 2022Article
- Article
- Safe and efficientMolecular therapy. Methods & clinical development · 2022Article
- Hematopoietic stem cell mobilization strategies to support high-dose chemotherapy: A focus on relapsed/refractory germ cell tumors.World journal of clinical oncology · 2021Review
Corrections and comments
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Authors and funding
11 authors at 4 institutions in 2 countries.
Funding
Abstract
We have developed an in vivo hemopoietic stem cell (HSC) gene therapy approach without the need for myelosuppressive conditioning and autologous HSC transplantation. It involves HSC mobilization and IV injection of a helper-dependent adenovirus HDAd5/35++ vector system. The current mobilization regimen consists of granulocyte colony-stimulating factor (G-CSF) injections over a 4-day period, followed by the administration of plerixafor/AMD3100. We tested a simpler, 2-hour, G-CSF-free mobilization regimen using truncated GRO-β (MGTA-145; a CXCR2 agonist) and plerixafor in the context of in vivo HSC transduction in mice. The MGTA-145+plerixafor combination resulted in robust mobilization of HSCs. Importantly, compared with G-CSF+plerixafor, MGTA-145+plerixafor led to significantly less leukocytosis and no elevation of serum interleukin-6 levels and was thus likely to be less toxic. With both mobilization regimens, after in vivo selection with O6-benzylguanine (O6BG)/BCNU, stable GFP marking was achieved in >90% of peripheral blood mononuclear cells. Genome-wide analysis showed random, multiclonal vector integration. In vivo HSC transduction after mobilization with MGTA-145+plerixafor in a mouse model for thalassemia resulted in >95% human γ-globin+ erythrocytes at a level of 36% of mouse β-globin. Phenotypic analyses showed a complete correction of thalassemia. The γ-globin marking percentage and level were maintained in secondary recipients, further demonstrating that MGTA145+plerixafor mobilizes long-term repopulating HSCs. Our study indicates that brief exposure to MGTA-145+plerixafor may be advantageous as a mobilization regimen for in vivo HSC gene therapy applications across diseases, including thalassemia and sickle cell disease.
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