Storage lesions

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Animals1

Interventions

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AAV1-GUSB gene therapy

downin mice1

1 study

AAV1-GUSB gene therapy prevents storage lesions in mice.
“completely prevented the development of storage lesions throughout the central nervous system (CNS).”
Animalsin MPS VII micesingle adeno-associated virus serotype 1 vector injection into the ventricle at 15.5 days of gestationcentral nervous system
Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII
Molecular therapy : the journal of the American Society of Gene Therapy19 Apr 2006

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