Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai
Patent
1998-10-29
2000-05-23
Campell, Bruce R.
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Carbohydrate doai
435208, 4353201, 435325, 435183, 424 931, 424 9461, A01N 4304
Patent
active
060666269
ABSTRACT:
The present invention provides recombinant viral and non-viral vectors comprising a transgene encoding a biologically active human lysosomal enzyme that are able to infect and/or transfect and sustain expression of the biologically active human lysosomal enzyme transgene in mammalian cells deficient therein. In addition, methods are provided for providing a biologically active human lysosomal enzyme to cells deficient therein, which comprises introducing into the cells a vector comprising and expressing a transgene encoding the biologically active human lysosomal enzyme, wherein the vector is taken up by the cells, the transgene is expressed and biologically active enzyme is produced. The cells may be infected and/or transfected by the vector, dependent upon whether the vector is a viral vector and/or plasmid or the like. The invention also provides a method of supplying a biologically active human lysosomal enzyme to other distant cells deficient therein wherein the transfected and/or infected cells harboring the vector secrete the biologically active enzyme which is then taken up by the other deficient cells. In a preferred embodiment the present invention provides for sustained production of biologically human active .alpha.-galactosidase A in cells of Fabry individuals that are deficient in said enzyme.
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Cheng Seng H.
Yew Nelson S.
Ziegler Robin J.
Campell Bruce R.
Genzyme Corporation
Lazar Steven R.
Shukla Ram
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