In utero gene transfer to the mouse nervous system

Ahad A. Rahim, Andrew M.S. Wong, Suzanne M.K. Buckley, Jerry K.Y. Chan, Anna L. David, Jonathan D. Cooper, Charles Coutelle, Donald M. Peebles, Simon N. Waddington

Research output: Contribution to journalArticlepeer-review

10 Scopus citations


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 themid-1980s. Recently, improvements in vector design and production have culminated in near-complete correction of a mousemodel of spinal muscular atrophy. In the present article, we review perinatal gene transfer from both a therapeutic and technological perspective.

Original languageEnglish
Pages (from-to)1489-1493
Number of pages5
JournalBiochemical Society transactions
Issue number6
StatePublished - Dec 2010


  • Gene therapy
  • In utero gene transfer
  • Lysosomal storage disorder
  • Mouse nervous system
  • Viral vector


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