GENE THERAPY FOR GAUCHER'S DISEASE.

摘要

The use of a vector selected from the group of adenovirus, adeno-associated virus (VAA), vaccines, herpes virus, bacteriophages, cosmids, fungal vectors for the preparation of a medicament for treating lysosomal storage disease, where the disease by Lysosomal storage is Gaucher's disease where the vector comprises and expresses a transgene encoding a biologically active human a-glucosidase, the vector is admitted in vivo by the target cells, the transgene is expressed therein and the enzyme is produced biologically active in vivo.