ArticleHuman gene therapy methods2013
Human miR223 promoter as a novel myelo-specific promoter for chronic granulomatous disease gene therapy.
Article in Human gene therapy methods, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 23 citations in OpenAlex.
- Gene editing of NCF1 loci is associated with homologous recombination and chromosomal rearrangements.Communications biology · 2024Article
- CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.Frontiers in immunology · 2022Article
- Tanshinone IIA and Astragaloside IV Inhibit miR-223/JAK2/STAT1 Signalling Pathway to Alleviate Lipopolysaccharide-Induced Damage in Nucleus Pulposus Cells.Disease markers · 2021Article
- Gene Therapy for Primary Immunodeficiency.HemaSphere · 2021Review
- Differential Transgene Silencing of Myeloid-Specific Promoters in theHuman gene therapy · 2020Article
- Lentiviral gene therapy vector with UCOE stably restores function in iPSC-derived neutrophils of a CDG patient.Matters · 2018Article
- TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis.Scientific reports · 2017Article
- CRISPR/Cas9-generated p47Scientific reports · 2017Article
- CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells.Experimental hematology · 2015Article
- Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Lentiviral vectors containing mouse Csf1r control elements direct macrophage-restricted expression in multiple species of birds and mammals.Molecular therapy. Methods & clinical development · 2014Article
- Gene therapy on the move.EMBO molecular medicine · 2013Review
Corrections and comments
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Authors and funding
13 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Targeting transgene expression to specific hematopoietic cell lineages could contribute to the safety of retroviral vectors in gene therapeutic applications. Chronic granulomatous disease (CGD), a defect of phagocytic cells, can be managed by gene therapy, using retroviral vectors with targeted expression to myeloid cells. In this context, we analyzed the myelospecificity of the human miR223 promoter, which is known to be strongly upregulated during myeloid differentiation, to drive myeloid-restricted expression of p47(phox) and gp91(phox) in mouse models of CGD and in primary patient-derived cells. The miR223 promoter restricted the expression of p47(phox), gp91(phox), and green fluorescent protein (GFP) within self-inactivating (SIN) gamma- and lentiviral vectors to granulocytes and macrophages, with only marginal expression in lymphocytes or hematopoietic stem and progenitor cells. Furthermore, gene transfer into primary CD34+ cells derived from a p47(phox) patient followed by ex vivo differentiation to neutrophils resulted in restoration of Escherichia coli killing activity by miR223 promoter-mediated p47(phox) expression. These results indicate that the miR223 promoter as an internal promoter within SIN gene therapy vectors is able to efficiently correct the CGD phenotype with negligible activity in hematopoietic progenitors, thereby limiting the risk of insertional oncogenesis and development of clonal dominance.
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