Ontology highlight
ABSTRACT:
SUBMITTER: Ferla R
PROVIDER: S-EPMC3581049 | biostudies-literature | 2013 Feb
REPOSITORIES: biostudies-literature
Ferla Rita R O'Malley Thomas T Calcedo Roberto R O'Donnell Patricia P Wang Ping P Cotugno Gabriella G Claudiani Pamela P Wilson James M JM Haskins Mark M Auricchio Alberto A
Human gene therapy 20130130 2
Liver gene transfer with adeno-associated viral (AAV) 2/8 vectors is being considered for therapy of systemic diseases like mucopolysaccharidosis type VI (MPS VI), a lysosomal storage disease due to deficiency of arylsulfatase B (ARSB). We have previously reported that liver gene transfer with AAV2/8 results in sustained yet variable expression of ARSB. We hypothesized that the variability we observed could be due to pre-existing immunity to wild-type AAV8. To test this, we compared the levels o ...[more]