In utero gene transfer to the mouse nervous system.
Biochem Soc Trans
; 38(6): 1489-93, 2010 Dec.
Article
em En
| MEDLINE
| ID: mdl-21118113
ABSTRACT
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças por Armazenamento dos Lisossomos
/
Técnicas de Transferência de Genes
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Feto
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Sistema Nervoso
Limite:
Animals
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Female
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Humans
/
Pregnancy
Idioma:
En
Revista:
Biochem Soc Trans
Ano de publicação:
2010
Tipo de documento:
Article
País de afiliação:
Reino Unido