ArticleGene therapy2005
Human artificial chromosome (HAC) vector provides long-term therapeutic transgene expression in normal human primary fibroblasts.
Article in Gene therapy, 2005. 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, 62 citations in OpenAlex.
- Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes.Cellular and molecular life sciences : CMLS · 2021Review
- Technique of laser chromosome welding for chromosome repair and artificial chromosome creation.Biomedical optics express · 2018Article
- Multiple expression cassette exchange via TP901-1, R4, and Bxb1 integrase systems on a mouse artificial chromosome.FEBS open bio · 2017Article
- The soluble form of BMPRIB is a novel therapeutic candidate for treating bone related disorders.Scientific reports · 2016Article
- Retargeting of microcell fusion towards recipient cell-oriented transfer of human artificial chromosome.BMC biotechnology · 2015Article
- Human antibody production in transgenic animals.Archivum immunologiae et therapiae experimentalis · 2015Review
- A pathway from chromosome transfer to engineering resulting in human and mouse artificial chromosomes for a variety of applications to bio-medical challenges.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2015Review
- De novo formed satellite DNA-based mammalian artificial chromosomes and their possible applications.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2015Review
- A new generation of human artificial chromosomes for functional genomics and gene therapy.Cellular and molecular life sciences : CMLS · 2013Review
- Human artificial chromosomes for gene delivery and the development of animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- Application of a bacterial artificial chromosome modification system for a human artificial chromosome vector.Yonago acta medica · 2011Article
- A method for producing transgenic cells using a multi-integrase system on a human artificial chromosome vector.PloS one · 2011Article
- Article
- Prospects for the use of artificial chromosomes and minichromosome-like episomes in gene therapy.Journal of biomedicine & biotechnology · 2010Article
- Introduction of a CD40L genomic fragment via a human artificial chromosome vector permits cell-type-specific gene expression and induces immunoglobulin secretion.Journal of human genetics · 2008Article
- Modeling chromosomes in mouse to explore the function of genes, genomic disorders, and chromosomal organization.PLoS genetics · 2006Review
- Exogenous gene expression and growth regulation of hematopoietic cells via a novel human artificial chromosome.Journal of human genetics · 2006Article
- Human artificial chromosome vectors meet stem cells: new prospects for gene delivery.Stem cell reviews · 2006Review
- Artificial and engineered chromosomes: developments and prospects for gene therapy.Chromosoma · 2005Review
- A novel expression system for genomic DNA loci using a human artificial chromosome vector with transformation-associated recombination cloning.Journal of human genetics · 2005Article
Corrections and comments
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Human artificial chromosomes (HACs) segregating freely from host chromosomes are potentially useful to ensure both safety and duration of gene expression in therapeutic gene delivery. However, low transfer efficiency of intact HACs to the cells has hampered the studies using normal human primary cells, the major targets for ex vivo gene therapy. To elucidate the potential of HACs to be vectors for gene therapy, we studied the introduction of the HAC vector, which is reduced in size and devoid of most expressed genes, into normal primary human fibroblasts (hPFs) with microcell-mediated chromosome transfer (MMCT). We demonstrated the generation of cytogenetically normal hPFs harboring the structurally defined and extra HAC vector. This introduced HAC vector was retained stably in hPFs without translocation of the HAC on host chromosomes. We also achieved the long-term production of human erythropoietin for at least 12 weeks in them. These results revealed the ability of HACs as novel options to circumvent issues of conventional vectors for gene therapy.
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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.