Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

Caroline Sevin,Lucie Verot,Abdellatif Benraiss,D. Van Dam,Delphine Bonnin,Guy Nagels,Françoise Fouquet,Volkmar Gieselmann,Marie T. Vanier,P.P. De Deyn,Patrick Aubourg,Nathalie Cartier

Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

2007

Caroline Sevin
Lucie Verot
Abdellatif Benraiss
D. Van Dam
Delphine Bonnin
Guy Nagels
Françoise Fouquet
Volkmar Gieselmann
Marie T. Vanier
P.P. De Deyn
Patrick Aubourg
Nathalie Cartier

Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

Keywords:

Virus
Adeno-associated virus
Parvoviridae
Degenerative disease
Virology
Genetic enhancement
Metachromatic leukodystrophy
Lysosomal storage disease
Pathology
Biology
Sphingolipidosis
genetic transfer
Immunology
Galactocerebroside
Arylsulfatase A
Knockout mouse

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