Article
Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.
Human gene therapy - 1 Feb 2013
Ferla Rita, O'Malley Thomas, Calcedo Roberto, O'Donnell Patricia, Wang Ping, Cotugno Gabriella, Claudiani Pamela, Wilson James M, Haskins Mark, Auricchio Alberto
Abstract excerpt
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...
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