FIELD: biotechnology.
SUBSTANCE: invention refers to biotechnology and molecular biology. Disclosed is a recombinant adeno-associated virus (rAAV) particle for use in the treatment of Type I Mucopolysaccharidosis (MPS I), having AAV-capsid and having 5' inverted end repeat (ITR) packed therein, human alpha-L-Iduronidase gene (hIDUA) under control of regulatory sequences which control its expression, and 3' ITR AAV, where said hIDUA gene has a sequence shown in SEQ ID NO: 1, which codes functional alpha-L-iduronidase of a human, as well as a composition and use.
EFFECT: invention can be applied in medicine.
13 cl, 2 dwg, 4 ex
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