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 the mid-1980s. Recently, improvements in vector design and production have culminated in near-complete correction of a mouse model of spinal muscular atrophy. In the present article, we review perinatal gene transfer from both a therapeutic and technological perspective.
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December 2010
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Conference Article|
November 24 2010
In utero gene transfer to the mouse nervous system
Ahad A. Rahim;
Ahad A. Rahim
*Institute for Women's Health, University College London, 86–96 Chenies Mews, London WC1E 6HX, U.K.
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Andrew M.S. Wong;
Andrew M.S. Wong
†Paediatric Storage Disease Laboratory, Institute of Psychiatry, Kings College London, 125 Coldharbour Lane, London SE5 9NU, U.K.
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Suzanne M.K. Buckley;
Suzanne M.K. Buckley
‡Department of Haematology, University College Medical School, Rowland Hill Street, London NW3 2PF, U.K.
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Jerry K.Y. Chan;
Jerry K.Y. Chan
§Division of Maternal and Fetal Medicine, Experimental Fetal Medicine Group, Department of Obstetrics and Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore
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Anna L. David;
Anna L. David
*Institute for Women's Health, University College London, 86–96 Chenies Mews, London WC1E 6HX, U.K.
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Jonathan D. Cooper;
Jonathan D. Cooper
†Paediatric Storage Disease Laboratory, Institute of Psychiatry, Kings College London, 125 Coldharbour Lane, London SE5 9NU, U.K.
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Charles Coutelle;
Charles Coutelle
∥National Heart and Lung Institute, Imperial College London, South Kensington, London SW7 2AZ, U.K.
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Donald M. Peebles;
Donald M. Peebles
*Institute for Women's Health, University College London, 86–96 Chenies Mews, London WC1E 6HX, U.K.
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Simon N. Waddington
Simon N. Waddington
1
*Institute for Women's Health, University College London, 86–96 Chenies Mews, London WC1E 6HX, U.K.
1To whom correspondence should be addressed (email s.waddington@medsch.ucl.ac.uk).
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Publisher: Portland Press Ltd
Received:
June 15 2010
Online ISSN: 1470-8752
Print ISSN: 0300-5127
© The Authors Journal compilation © 2010 Biochemical Society
2010
Biochem Soc Trans (2010) 38 (6): 1489–1493.
Article history
Received:
June 15 2010
Citation
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; In utero gene transfer to the mouse nervous system. Biochem Soc Trans 1 December 2010; 38 (6): 1489–1493. doi: https://doi.org/10.1042/BST0381489
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