ArticleGene therapy2008
Correction of a genetic defect in multipotent germline stem cells using a human artificial chromosome.
Article in Gene therapy, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
19 citing papers in PubMed, 36 citations in OpenAlex.
- Down syndrome-related transient abnormal myelopoiesis is attributed to a specific erythro-megakaryocytic subpopulation withHaematologica · 2021Article
- Evaluation of a Small Animal Irradiation System for Animal Experiments Using EBT3 Model GAFCHROMIC™ Film.The Kobe journal of medical sciences · 2018Article
- Mouse embryonic stem cells with a multi-integrase mouse artificial chromosome for transchromosomic mouse generation.Transgenic research · 2015Article
- Generation of a conditionally self-eliminating HAC gene delivery vector through incorporation of a tTAVP64 expression cassette.Nucleic acids research · 2015Article
- 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 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
- A novel transchromosomic system: stable maintenance of an engineered Mb-sized human genomic fragment translocated to a mouse chromosome terminal region.Transgenic research · 2014Article
- Re-engineering an alphoid(tetO)-HAC-based vector to enable high-throughput analyses of gene function.Nucleic acids research · 2013Article
- A new generation of human artificial chromosomes for functional genomics and gene therapy.Cellular and molecular life sciences : CMLS · 2013Review
- Autologous pluripotent stem cells generated from adult mouse testicular biopsy.Stem cell reviews and reports · 2012Article
- Human artificial chromosomes for gene delivery and the development of animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- De novo assembly of potential linear artificial chromosome constructs capped with expansive telomeric repeats.Plant methods · 2011Article
- Refined human artificial chromosome vectors for gene therapy and animal transgenesis.Gene therapy · 2011Article
- Article
- Human artificial chromosome with a conditional centromere for gene delivery and gene expression.DNA research : an international journal for rapid publication of reports on genes and genomes · 2010Article
- Localization of an hTERT repressor region on human chromosome 3p21.3 using chromosome engineering.Genome integrity · 2010Article
- Complete genetic correction of ips cells from Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Progress and prospects: human artificial chromosomes.Gene therapy · 2009Review
- A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
17 authors at 5 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Human artificial chromosomes (HACs) have several advantages as gene therapy vectors, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including regulatory elements. Multipotent germline stem (mGS) cells have a great potential for gene therapy because they can be generated from an individual's testes, and when reintroduced can contribute to the specialized function of any tissue. As a proof of concept, we herein report the functional restoration of a genetic deficiency in mouse p53-/- mGS cells, using a HAC with a genomic human p53 gene introduced via microcell-mediated chromosome transfer. The p53 phenotypes of gene regulation and radiation sensitivity were complemented by introducing the p53-HAC and the cells differentiated into several different tissue types in vivo and in vitro. Therefore, the combination of using mGS cells with HACs provides a new tool for gene and cell therapies. The next step is to demonstrate functional restoration using animal models for future gene therapy.
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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.