To provide a recombinant viral vector and a non-viral vector, comprising a transgene encoding a biologically active human lysosomal enzyme.
The vector, comprising the biologically active human lysosomal enzyme, that can infect and/or transfect with a cell deficient in the human lysosomal enzyme and sustainably express the transgene of the biologically active human lysosomal enzyme, is provided to a cell deficient therein. Further, the biologically active human lysosomal enzyme is provided to the other distant cell deficient therein. The transfected and/or infected cell having the vector secretes a biologically active enzyme, which is then taken in the other deficient cell. In a preferable concrete example, a biologically active human -galactosidase A is sustainably produced in a cell of Fabry disease, deficient in the enzyme.
ZIEGLER ROBIN J
CHENG SENG H
Hiroshi Yamazaki
Tadayuki Motoyama
Kenji Tomita