FIELD: medicine.
SUBSTANCE: invention relates to the treatment of symptoms of type II mucopolysaccharidosis. A method of preventing, inhibiting, or treating one or more symptoms of type II mucopolysaccharidosis (MPS II) in a human includes intrathecal administration of a composition containing an effective amount of a recombinant adeno-associated virus (rAAV) vector containing an open reading frame encoding iduronate- 2-sulfatase to prevent, inhibit or treat one or more symptoms of MPS II, wherein rAAV is either rAAV9 or rAAVrh10.
EFFECT: expansion of the arsenal of agents for a specific purpose.
17 cl, 20 dwg, 3 ex
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