Journal of Pediatric Surgery
Volume 40, Issue 12 , Pages 1817-1821 , December 2005

Lentiviral vector–mediated, in vivo gene transfer to the tracheobronchial tree in fetal rabbits

  • Erik D. Skarsgard

      Affiliations

    • Division of Pediatric General Surgery, British Columbia Children's Hospital, Vancouver, Canada, BC V6H 3V4
    • Corresponding Author InformationCorresponding author. Tel.: +1 604 875 3744; fax: +1 604 875 2721.
    • ,
  • Louis Huang

      Affiliations

    • Division of Pediatric General Surgery, British Columbia Children's Hospital, Vancouver, Canada, BC V6H 3V4
    • ,
  • Stacy C. Reebye

      Affiliations

    • Division of Pediatric General Surgery, British Columbia Children's Hospital, Vancouver, Canada, BC V6H 3V4
    • ,
  • Adam Y. Yeung

      Affiliations

    • Division of Pediatric General Surgery, British Columbia Children's Hospital, Vancouver, Canada, BC V6H 3V4
    • ,
  • William W. Jia

      Affiliations

    • Division of Neurosurgery, Department of Surgery, Brain Research Centre, University of British Columbia, Vancouver, Canada V6T 2B5

References 

  1. Ratjen F, Doring G. Cystic fibrosis. Lancet. 2003;361:681–689
  2. Griesenbach U, Geddes DM, Alton EW. Advances in cystic fibrosis gene therapy. Curr Opin Pulm Med. 2004;10:542–546
  3. Husang L, Reebye SC, Yeung AY, et al. Lentiviral-mediated fetal gene therapy for monogenic disorders: development of an in vitro rabbit model. Fetal Diagn Ther (in press).
  4. Chauhan DP, Srivastava AS, Moustafa ME, et al. In utero gene therapy: prospect and future. Curr Pharm Des. 2004;10:3663–3672
  5. Cystic Fibrosis Foundation . Patient Registry 2003 Annual Report. Maryland: Bethesda; 2003;
  6. Monahan PE, Samulski RJ. AAV vectors: is clinical success on the horizon?. Gene Ther. 2000;7:24–30
  7. Williams DA, Smith FO. Progress in the use of gene transfer methods to treat genetic blood diseases. Hum Gene Ther. 2000;11:2059–2066
  8. Raper SE, Yudkoff M, Chirmule N, et al. A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther. 2002;13:163–175
  9. Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003;80:148–158
  10. Cockrell AS, Kafri T. HIV-1 vectors: fulfillment of expectations, further advancements, and still a way to go. Curr HIV Res. 2003;1:419–439
  11. Trono D. Lentiviral vectors: turning a deadly foe into a therapeutic agent. Gene Ther. 2000;7:20–23
  12. Lim FY, Kobinger GP, Weiner DJ, et al. Human fetal trachea-SCID mouse xenografts: efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer. J Pediatr Surg. 2003;38:834–839
  13. Cereseto A, Giacca M. Integration site selection by retroviruses. AIDS Rev. 2004;6:13–21
  14. Coutelle C, Rodeck C. On the scientific and ethical issues of fetal somatic gene therapy. Gene Ther. 2002;9:670–673

 Presented at the 38th Annual Meeting of the Pacific Association of Pediatric Surgeons, May 22-26, 2005, Vancouver, Canada.

PII: S0022-3468(05)00676-7

doi: 10.1016/j.jpedsurg.2005.08.047

Journal of Pediatric Surgery
Volume 40, Issue 12 , Pages 1817-1821 , December 2005

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