ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 24 citations in OpenAlex.
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- CD90-targeted lentiviral vectors for HSC gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Unmasking the Deceptive Nature of Cancer Stem Cells: The Role of CD133 in Revealing Their Secrets.International journal of molecular sciences · 2023Review
- Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Implications of hematopoietic stem cells heterogeneity for gene therapies.Gene therapy · 2021Review
- Baboon Envelope Pseudotyped "Nanoblades" Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34Frontiers in genome editing · 2021Article
- Gene therapy of hematological disorders: current challenges.Gene therapy · 2019Review
- In Vivo Hematopoietic Stem Cell Transduction.Hematology/oncology clinics of North America · 2017Review
- Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.The Journal of clinical investigation · 2016Article
- Optimizing autologous cell grafts to improve stem cell gene therapy.Experimental hematology · 2016Review
- Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Blood · 2016Review
- Production of lentiviral vectors.Molecular therapy. Methods & clinical development · 2016Review
- Chemoprotection of murine hematopoietic cells by combined gene transfer of cytidine deaminase (CDD) and multidrug resistance 1 gene (MDR1).Journal of experimental & clinical cancer research : CR · 2015Article
- CD105 is a surface marker for receptor-targeted gene transfer into human long-term repopulating hematopoietic stem cells.Stem cells and development · 2015Article
Corrections and comments
- Erratum issued
Authors and funding
15 authors at 5 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy for hematological disorders relies on the genetic modification of CD34(+) cells, a heterogeneous cell population containing about 0.01% long-term repopulating cells. Here, we show that the lentiviral vector CD133-LV, which uses a surface marker on human primitive hematopoietic stem cells (HSCs) as entry receptor, transfers genes preferentially into cells with high engraftment capability. Transduction of unstimulated CD34(+) cells with CD133-LV resulted in gene marking of cells with competitive proliferative advantage in vitro and in immunodeficient mice. The CD133-LV-transduced population contained significantly more cells with repopulating capacity than cells transduced with vesicular stomatitis virus (VSV)-LV, a lentiviral vector pseudotyped with the vesicular stomatitis virus G protein. Upon transfer of a barcode library, CD133-LV-transduced cells sustained gene marking in vivo for a prolonged period of time with a 6.7-fold higher recovery of barcodes compared to transduced control cells. Moreover, CD133-LV-transduced cells were capable of repopulating secondary recipients. Lastly, we show that this targeting strategy can be used for transfer of a therapeutic gene into CD34(+) cells obtained from patients suffering of X-linked chronic granulomatous disease. In conclusion, direct gene transfer into CD133(+) cells allows for sustained long-term engraftment of gene corrected cells.
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