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Publication Detail
The current status and future directions of fetal gene therapy
  • Publication Type:
    Journal article
  • Publication Sub Type:
    JOUR
  • Authors:
    David AL, Themis M, Waddington SN, Gregory L, Buckley SMK, Nivsarkar M, Cook T, Peebles D, Rodeck CH, Coutelle C
  • Publication date:
    2003
  • Pagination:
    181, 209
  • Volume:
    7
  • Notes:
    Application of gene therapy in utero has been considered as a strategy for treatment or even prevention of early onset genetic disorders such as cystic fibrosis and Duchenne muscular dystrophy. Prenatal gene transfer may target rapidly expanding stem cell populations that are inaccessible after birth, permit induction of immune tolerance against vector and transgene and allow permanent gene transfer by use of integrating vector systems. Application of this therapy in the fetus must be safe, reliable and cost-effective. Recent developments in the understanding of genetic disease, vector design, and minimally invasive delivery techniques have brought fetal gene therapy closer to clinical practice. Prenatal studies in animal models are being pursued in parallel with adult studies of gene therapy, but they remain presently at the experimental stage.
Abstract
Application of gene therapy in utero has been considered as a strategy for treatment or even prevention of early onset genetic disorders such as cystic fibrosis and Duchenne muscular dystrophy. Prenatal gene transfer may target rapidly expanding stem cell populations that are inaccessible after birth, permit induction of immune tolerance against vector and transgene and allow permanent gene transfer by use of integrating vector systems. Application of this therapy in the fetus must be safe, reliable and cost-effective. Recent developments in the understanding of genetic disease, vector design, and minimally invasive delivery techniques have brought fetal gene therapy closer to clinical practice. Prenatal studies in animal models are being pursued in parallel with adult studies of gene therapy, but they remain presently at the experimental stage.
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