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Publication Detail
The case for intrauterine gene therapy
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Journal Article
  • Authors:
    Mattar CN, Waddington SN, Biswas A, Davidoff AM, Choolani M, Chan JKY, Nathwani AC
  • Publication date:
  • Pagination:
    697, 709
  • Journal:
    Best Practice and Research: Clinical Obstetrics and Gynaecology
  • Volume:
  • Issue:
  • Status:
  • Print ISSN:
Single-gene disorders can cause perinatal mortality or severe permanent morbidity. Intrauterine gene therapy seeks to correct the genetic defect in the early stages of pathogenesis through delivery of a vector system expressing the therapeutic transgene to the fetus. Advantages of intrauterine gene therapy include prevention of irreversible organ damage, potentially inducing central tolerance and wider bio-distribution, including the brain after delivery of vector. Already, proof-of-cure has been demonstrated in knockout animal models for several diseases. Long-term outcomes pertaining to efficacy and durability of transgene expression and safety are under investigation in clinically relevant non-human primate models. Bystander effects in the mother from transplacental vector trafficking require further assessment. In this chapter, we discuss the candidate diseases amenable to intrauterine gene therapy, current state-of-the-art evidence, and potential clinical applications. © 2012 Elsevier Ltd. All rights reserved.
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