CPC A61K 38/47 (2013.01) [C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2830/007 (2013.01); C12Y 302/01076 (2013.01)] | 25 Claims |
1. A method to treat one or more symptoms of mucopolysaccharidosis type II (MPSII) in a human having MPSII, consisting of: administering to a cisterna magna of the human a composition comprising an amount of a recombinant adeno-associated virus (rAAV) 9 or rAAVrh10 vector comprising an open reading frame encoding iduronate-2-sulfatase in a physiologically compatible buffer effective to treat the one or more symptoms of MPSII.
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