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In utero gene transfer to the mouse nervous system

Rahim, AA; Wong, AMS; Buckley, SMK; Chan, JKY; David, AL; Cooper, JD; Coutelle, C; ... Waddington, SN; + view all (2010) In utero gene transfer to the mouse nervous system. BIOCHEM SOC T , 38 1489 - 1493. 10.1042/BST0381489.

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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 ell 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 art! le we review perinatal gene transfer from both a therapeutic and technological perspective

Type: Article
Title: In utero gene transfer to the mouse nervous system
DOI: 10.1042/BST0381489
Keywords: gene therapy, in utero gene transfer, lysosomal storage disorder, mouse nervous system, viral vector, LEBERS CONGENITAL AMAUROSIS, CEREBRAL-CORTEX, CELL LINEAGE, DELIVERY, THERAPY, FETAL, TRANSDUCTION, VECTOR, ADULT, BRAIN
URI: http://discovery.ucl.ac.uk/id/eprint/397465
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