ArticleGene therapy2011
Refined human artificial chromosome vectors for gene therapy and animal transgenesis.
Article in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.
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Who cites it
62 citing papers in PubMed, 110 citations in OpenAlex.
- Identification of TMEM115 as a tumor suppressor gene and clinical prognostic factor that influences TERT expression in pancreatic cancer.Scientific reports · 2026Article
- The design and engineering of synthetic genomes.Nature reviews. Genetics · 2025Review
- The lives of cells, recorded.Nature reviews. Genetics · 2025Review
- Microcell-mediated chromosome transfer between non-identical human iPSCs.Molecular therapy. Nucleic acids · 2024Article
- Treatment of CHO cells with Taxol and reversine improves micronucleation and microcell-mediated chromosome transfer efficiency.Molecular therapy. Nucleic acids · 2023Article
- Human artificial chromosome carrying 3p21.3-p22.2 region suppresses hTERT transcription in oral cancer cells.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2023Article
- Full-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model.Scientific reports · 2023Article
- Simultaneous loading of PCR-based multiple fragments on mouse artificial chromosome vectors in DT40 cell for gene delivery.Scientific reports · 2022Article
- Panel of human cell lines with human/mouse artificial chromosomes.Scientific reports · 2022Article
- A transchromosomic rat model with human chromosome 21 shows robust Down syndrome features.American journal of human genetics · 2022Article
- Highly Efficient Microcell-Mediated Transfer of HACs Containing a Genomic Region of Interest into Mammalian Cells.Current protocols · 2021Article
- Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling.Molecular therapy. Nucleic acids · 2021Article
- Blastocyst complementation using Prdm14-deficient rats enables efficient germline transmission and generation of functional mouse spermatids in rats.Nature communications · 2021Article
- Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes.Cellular and molecular life sciences : CMLS · 2021Review
- Development of Caco-2 cells expressing four CYPs via a mammalian artificial chromosome.BMC biotechnology · 2020Article
- Review
- A non-mosaic transchromosomic mouse model of down syndrome carrying the long arm of human chromosome 21.eLife · 2020Article
- Human AlphoidCells · 2020Article
- An efficient protein production system via gene amplification on a human artificial chromosome and the chromosome transfer to CHO cells.Scientific reports · 2019Article
- Humanized UGT2 and CYP3A transchromosomic rats for improved prediction of human drug metabolism.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
2 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
23 authors at 6 institutions in 4 countries.
Funding
Abstract
Human artificial chromosomes (HACs) have several advantages as gene therapy vectors, including stable episomal maintenance, and the ability to carry large gene inserts. We previously developed HAC vectors from the normal human chromosomes using a chromosome engineering technique. However, endogenous genes were remained in these HACs, limiting their therapeutic applications. In this study, we refined a HAC vector without endogenous genes from human chromosome 21 in homologous recombination-proficient chicken DT40 cells. The HAC was physically characterized using a transformation-associated recombination (TAR) cloning strategy followed by sequencing of TAR-bacterial artificial chromosome clones. No endogenous genes were remained in the HAC. We demonstrated that any desired gene can be cloned into the HAC using the Cre-loxP system in Chinese hamster ovary cells, or a homologous recombination system in DT40 cells. The HAC can be efficiently transferred to other type of cells including mouse ES cells via microcell-mediated chromosome transfer. The transferred HAC was stably maintained in vitro and in vivo. Furthermore, tumor cells containing a HAC carrying the suicide gene, herpes simplex virus thymidine kinase (HSV-TK), were selectively killed by ganciclovir in vitro and in vivo. Thus, this novel HAC vector may be useful not only for gene and cell therapy, but also for animal transgenesis.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.