Therapeutic gene targeting View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

1998-02-01

AUTHORS

RJ Yáñez, ACG Porter

ABSTRACT

Gene targeting is the use of homologous recombination to make defined alterations to the genome. One of the possible outcomes of gene targeting is the accurate correction of genetic defects, and this would make it the ideal method of gene therapy for single gene disorders. While gene targeting has been achieved both in human cell lines and in nontransformed, primary human cells, its low efficiency has been a major limitation to its therapeutic potential. Gene therapy by in vivo gene targeting is therefore impractical without dramatic improvements in targeting efficiency. Ex vivo approaches might more realistically be considered, but would benefit from progress in the isolation and growth of somatic stem cells and improvements in targeting efficiency. We provide here a brief review of the challenges of gene therapy by gene targeting. This is followed by a critical overview of recent developments in gene targeting techniques, and in our understanding of the underlying processes of homologous and nonhomologous recombination. More... »

PAGES

149-159

References to SciGraph publications

  • 1992-12. Targeted breakage of a human chromosome mediated by cloned human telomeric DNA in NATURE GENETICS
  • 1989-03. Production of chimaeric mice containing embryonic stem (ES) cells carrying a homoeobox Hox 1.1 allele mutated by homologous recombination in NATURE
  • 1993-07. Cloning of human, mouse and fission yeast recombination genes homologous to RAD51 and recA in NATURE GENETICS
  • 1996-11. PCR-based gene targeting of the inducible nitric oxide synthase (NOS2) locus in murine ES cells, a new and more cost-effective approach in TRANSGENIC RESEARCH
  • 1985-09. Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination in NATURE
  • 1997-05. A sense-abl response? in NATURE
  • 1997-05. Interaction between ATM protein and c-Abl in response to DNA damage in NATURE
  • 1997-07. Activity of DNA ligase IV stimulated by complex formation with XRCC4 protein in mammalian cells in NATURE
  • 1997-04. Embryonic lethality and radiation hypersensitivity mediated by Rad51 in mice lacking Brca2 in NATURE
  • 1994-01. Gene targeting in normal somatic cells: inactivation of the interferon–γ receptor in myoblasts in NATURE GENETICS
  • 1993-06. Mismatch recognition in chromosomal interactions and speciation in CHROMOSOMA
  • 1994-03. Gene conversion in the chicken immunoglobulin locus: A paradigm of homologous recombination in higher eukaryotes in CELLULAR AND MOLECULAR LIFE SCIENCES
  • 1996-03. RNA facilitates RecA-mediated DNA pairing and strand transfer between molecules bearing limited regions of homology in MOLECULAR GENETICS AND GENOMICS
  • 1996-02. Efficient modification of human chromosomal alleles using recombination-proficient chicken/human microcell hybrids in NATURE GENETICS
  • 1997-09-18. Gene therapy - promises, problems and prospects in NATURE
  • 1997-07-01. Efficient gene targeting in mouse embryonic stem cells in GENE THERAPY
  • 1997-04. Functional interaction between DNA-PK and c-Abl in response to DNA damage in NATURE
  • 1993-07. Targeting vector configuration and method of gene transfer influence targeted correction of theAPRT gene in Chinese hamster ovary cells in SOMATIC CELL AND MOLECULAR GENETICS
  • Journal

    TITLE

    Gene Therapy

    ISSUE

    2

    VOLUME

    5

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/sj.gt.3300601

    DOI

    http://dx.doi.org/10.1038/sj.gt.3300601

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1043341032

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/9578833


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