ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed.
- Review
- Improved functionality and potency of next generation BinMLV viral vectors toward safer gene therapy.Molecular therapy. Methods & clinical development · 2021Article
- Retroviral gene therapy in Germany with a view on previous experience and future perspectives.Gene therapy · 2021Review
- Immunotherapy with NK cells: recent developments in gene modification open up new avenues.Oncoimmunology · 2020Review
- Toward Combined Cell and Gene Therapy for Genodermatoses.Cold Spring Harbor perspectives in biology · 2020Review
- Proceedings From the First International Workshop at Sidra Medicine: "Engineered Immune Cells in Cancer Immunotherapy (EICCI): From Discovery to Off-the-Shelf Development", 15Frontiers in immunology · 2020Article
- Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application.Molecular therapy. Methods & clinical development · 2019Article
- Review
- High Cytotoxic Efficiency of Lentivirally and Alpharetrovirally Engineered CD19-Specific Chimeric Antigen Receptor Natural Killer Cells Against Acute Lymphoblastic Leukemia.Frontiers in immunology · 2019Article
- Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy.Molecular therapy. Nucleic acids · 2017Article
- Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells.Gene therapy · 2017Article
- Application of Droplet Digital PCR for Estimating Vector Copy Number States in Stem Cell Gene Therapy.Human gene therapy methods · 2016Article
- Towards a Safer, More Randomized Lentiviral Vector Integration Profile Exploring Artificial LEDGF Chimeras.PloS one · 2016Article
- An assessment of the effects of ectopic gp91phox expression in XCGD iPSC-derived neutrophils.Molecular therapy. Methods & clinical development · 2015Article
- Alpharetroviral vectors: from a cancer-causing agent to a useful tool for human gene therapy.Viruses · 2014Review
- Gene and cell therapy for children--new medicines, new challenges?Advanced drug delivery reviews · 2014Review
- Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.Genes · 2014Article
- Generation of X-CGD cells for vector evaluation from healthy donor CD34(+) HSCs by shRNA-mediated knock down of gp91(phox).Molecular therapy. Methods & clinical development · 2014Article
- Gene therapy on the move.EMBO molecular medicine · 2013Review
- Review
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Authors and funding
11 authors.
Funding
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Abstract
Comparative integrome analysis has revealed that the most neutral integration pattern among retroviruses is attributed to alpharetroviruses. We chose X-linked chronic granulomatous disease (X-CGD) as model to evaluate the potential of self-inactivating (SIN) alpharetroviral vectors for gene therapy of monogenic diseases. Therefore, we combined the alpharetroviral vector backbone with the elongation factor-1α short promoter, both considered to possess a low genotoxic profile, to drive transgene (gp91(phox)) expression. Following efficient transduction transgene expression was sustained and provided functional correction of the CGD phenotype in a cell line model at low vector copy number. Further analysis in a murine X-CGD transplantation model revealed gene-marking of bone marrow cells and oxidase positive granulocytes in peripheral blood. Transduction of human X-CGD CD34+ cells provided functional correction up to wild-type levels and long-term expression upon transplantation into a humanized mouse model. In contrast to lentiviral vectors, no aberrantly spliced transcripts containing cellular exons fused to alpharetroviral sequences were found in transduced cells, implying that the safety profile of alpharetroviral vectors may extend beyond their neutral integration profile. Taken together, this highlights the potential of this SIN alpharetroviral system as a platform for new candidate vectors for future gene therapy of hematopoietic disorders.
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