- Tytuł:
- Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer.
- Autorzy:
- Źródło:
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Gene therapy [Gene Ther] 2007 Mar; Vol. 14 (5), pp. 405-14. Date of Electronic Publication: 2006 Nov 09. - Typ publikacji:
- Journal Article; Research Support, Non-U.S. Gov't
- MeSH Terms:
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Brain /*enzymology
Dependovirus/*genetics
Genetic Therapy/*methods
Genetic Vectors/*administration & dosage
Leukodystrophy, Metachromatic/*therapy
Transduction, Genetic/*methods
Animals ;Brain Chemistry ; Cerebroside-Sulfatase/genetics ; Cerebroside-Sulfatase/metabolism ; Disease Models, Animal ; Galactosylceramides/analysis ; Galactosylceramides/metabolism ; Gangliosides/analysis ; Gangliosides/metabolism ; Genetic Vectors/genetics ; Immunohistochemistry ; Injections ; Leukodystrophy, Metachromatic/enzymology ; Mice ; Mice, Knockout ; Mice, Mutant Strains ; Motor Activity ; Treatment Failure
Czasopismo naukowe