ArticleGene therapy2016
BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector.
Article in Gene therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed, 13 citations in OpenAlex.
- Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia.The Journal of allergy and clinical immunology · 2024Article
- Future Drug Targets in Periodontal Personalised Medicine-A Narrative Review.Journal of personalized medicine · 2022Review
- A Single "All-in-One" Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair.Molecular therapy. Methods & clinical development · 2020Article
- DNA repair fidelity in stem cell maintenance, health, and disease.Biochimica et biophysica acta. Molecular basis of disease · 2020Review
- Bi-allelic Homology-Directed Repair with Helper-Dependent Adenoviruses.Molecular therapy. Methods & clinical development · 2019Article
- Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses.Molecular therapy. Methods & clinical development · 2019Article
- Gene Editing with Helper-Dependent Adenovirus Can Efficiently Introduce Multiple Changes Simultaneously over a Large Genomic Region.Molecular therapy. Nucleic acids · 2017Article
- Homology Requirements for Efficient, Footprintless Gene Editing at the CFTR Locus in Human iPSCs with Helper-dependent Adenoviral Vectors.Molecular therapy. Nucleic acids · 2016Article
- X-linked Agammaglobulinemia.Indian journal of pediatrics · 2016Review
- Immunohistochemical study of hair follicle stem cells in regenerated hair follicles induced by Wnt10b.International journal of medical sciences · 2016Article
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
X-linked agammaglobulinemia (XLA) is one of the most common humoral immunodeficiencies, which is caused by mutations in Bruton's tyrosine kinase (BTK) gene. To examine the possibility of using gene therapy for XLA, we constructed a helper-dependent adenovirus/adeno-associated virus BTK targeting vector (HD-Ad.AAV BTK vector) composed of a genomic sequence containing BTK exons 6-19 and a green fluorescence protein-hygromycin cassette driven by a cytomegalovirus promoter. We first used NALM-6, a human male pre-B acute lymphoblastic leukemia cell line, as a recipient to measure the efficiency of gene targeting by homologous recombination. We identified 10 clones with the homologous recombination of the BTK gene among 107 hygromycin-resistant stable clones isolated from two independent experiments. We next used cord blood CD34⁺ cells as the recipient cells for the gene targeting. We isolated colonies grown in medium containing cytokines and hygromycin. We found that the targeting of the BTK gene occurred in four of the 755 hygromycin-resistant colonies. Importantly, the gene targeting was also observed in CD19⁺ lymphoid progenitor cells that were differentiated from the homologous recombinant CD34⁺ cells during growth in selection media. Our study shows the potential for the BTK gene therapy using the HD-Ad.AAV BTK vector via homologous recombination in hematopoietic stem cells.
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