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Publication Detail
In utero gene transfer to the mouse nervous system.
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Journal Article
  • Authors:
    Rahim AA, Wong AMS, Buckley SMK, Chan JKY, David AL, Cooper JD, Coutelle C, Peebles DM, Waddington SN
  • Publication date:
  • Pagination:
    1489, 1493
  • Journal:
    Biochem Soc Trans
  • Volume:
  • Issue:
  • Status:
  • Country:
  • PII:
  • Language:
  • Keywords:
    Animals, Female, Fetus, Ganglia, Spinal, Gene Transfer Techniques, Genetic Vectors, Humans, Lysosomal Storage Diseases, Mice, Nervous System, Pregnancy
The cellular and molecular environment present in the fetus and early newborn provides an excellent opportunity for effective gene transfer. Innate and pre-existing anti-vector immunity may be attenuated or absent and the adaptive immune system predisposed to tolerance towards xenoproteins. Stem cell and progenitor cell populations are abundant, active and accessible. In addition, for treatment of early lethal genetic diseases of the nervous system, the overarching advantage may be that early gene supplementation prevents the onset of irreversible pathological changes. Gene transfer to the fetal mouse nervous system was achieved, albeit inefficiently, as far back as the mid-1980s. Recently, improvements in vector design and production have culminated in near-complete correction of a mouse model of spinal muscular atrophy. In the present article, we review perinatal gene transfer from both a therapeutic and technological perspective.
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