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Publication Detail
In Utero Gene Therapy: Current Challenges And Perspectives
  • Publication Type:
    Journal article
  • Publication Sub Type:
    JOUR
  • Authors:
    Waddington SN, Kramer MG, Hernandez-Alcoceba R, Buckley SMK, Themis M, Coutelle C, Prieto J
  • Publication date:
    2005
  • Pagination:
    661, 676
  • Volume:
    11
  • Issue:
    5
  • Notes:
    Over the past few years, considerable progress in prenatal diagnosis and surgery combined with improvements in vector design vindicate a reappraisal of the feasibility of in utero gene therapy for serious monogenetic diseases. As adult gene therapy gathers pace, several apparent obstacles to its application as a treatment may be overcome by pre- or early post-natal treatment. This review will examine the concepts and practice of prenatal vector administration. We aim to highlight the advantages of early therapeutic intervention focusing on diseases that could benefit greatly from a pre-natal gene therapy approach. We will pay special attention to the strategies and vectors which are most likely to be used for this application, and will speculate on their expected developments for the near future
Abstract
Over the past few years, considerable progress in prenatal diagnosis and surgery combined with improvements in vector design vindicate a reappraisal of the feasibility of in utero gene therapy for serious monogenetic diseases. As adult gene therapy gathers pace, several apparent obstacles to its application as a treatment may be overcome by pre- or early post-natal treatment. This review will examine the concepts and practice of prenatal vector administration. We aim to highlight the advantages of early therapeutic intervention focusing on diseases that could benefit greatly from a pre-natal gene therapy approach. We will pay special attention to the strategies and vectors which are most likely to be used for this application, and will speculate on their expected developments for the near future
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