FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and genetic engineering, in particular, to nucleic acids coding acid alpha-glucosidase (GAA). Present invention also relates to expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids coding acid alpha-glucosidase (GAA).
EFFECT: invention enables to use the invention as a gene therapy of individuals with Pompe disease and other diseases and disorders which can be treated using GAA.
86 cl, 21 dwg, 3 tbl, 14 ex
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