FIELD: medicine.
SUBSTANCE: disclosed is a method for preventing, inhibiting or treating one or more symptoms of type I (MPS I) mucopolysaccharidosis in humans, involving intrathecal introduction of a composition in need thereof to a human, containing an effective amount of a vector based on recombinant adeno-associated virus (rAAV) containing an open reading frame coding alpha-L-iduronidase, wherein rAAV is rAAV9 or rAAVrh10.
EFFECT: invention provides an effective method of preventing, inhibiting or treating one or more symptoms of MPS I in a human having the absence or deficiency associated with accumulations of a lysosomal enzyme.
17 cl, 20 dwg, 3 ex
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