AAV1-GUSB gene therapy

1. 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 Therapy·19 Apr 2006

More on Storage lesions

1. AAV1-GUSB gene therapy extends lifespan in mice.

   “However, the survival probability of the treated animals was significantly increased.”

   Animalsin MPS VII micesingle adeno-associated virus serotype 1 vector injection into the ventricle at 15.5 days of gestationbrain

   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 Therapy·19 Apr 2006