A METHOD FOR THE PRODUCTION OF A HUMAN PROTEIN IN A PLANT, IN PARTICULAR A HUMAN RECOMBINANT LYSOSOMAL ENZYME IN A CEREAL ENDOSPERM

摘要

Discloses a method for the production of recombinant human lysosomal enzyme in a cereal endosperm. The method comprises the construction of a plant expression vector comprising: (i) nucleotide sequence encoding a mature form of the human lysosomal enzyme; (ii) rice glutelin 4 promoter (GluB4pro) as an endosperm-specific promoter upstream the nucleotide sequence encoding said human lysosomal enzyme; (iii) leader known as LLTCK as a 5' UTR region; (iv) a PSGluB4 sequence encoding a signal peptide; and (v) a 3' UTR; The method further comprises a step of cereal plant transformation using the plant expression vector, whereby the human lysosomal enzyme is obtained and confined in an endosperm. Further discloses transgenic cereal plants transformed with the expression vector and the recovery of the recombinant lysosomal enzyme protein. Further discloses the use of the purified lysosomal enzyme for preparation of a medicament for use in treating certain conditions associated with lysosomal enzyme deficiency.