Plant cell culture expressing human lysosomal proteins and uses thereof

Abstract

A device, system and method for producing glycosylated proteins in plant culture, particularly proteins having a high mannose glycosylation, while targeting such proteins with an ER signal and/or by-passing the Golgi. The invention further relates to vectors and methods for expression and production of enzymatically active high mannose lysosomal enzymes using transgenic plant root, particularly carrot cells. More particularly, the invention relates to host cells, particularly transgenic suspended carrot cells, vectors and methods for high yield expression and production of biologically active high mannose Glucocerebrosidase (GCD). The invention further provides for compositions and methods for the treatment of lysosomal storage diseases.

What is claimed is: 
     
         1 . A method of treating a subject having Gaucher's disease comprising orally administering to said subject a therapeutically effective amount of lyophilized plant cells expressing a recombinant human glucocerebrosidase protein, thereby treating said Gaucher's disease in said subject. 
     
     
         2 . The method of  claim 1 , wherein said plant cells are carrot cells from carrot cell suspension culture. 
     
     
         3 . The method of  claim 2 , wherein said carrot cells are from a carrot cell suspension culture. 
     
     
         4 . The method of  claim 1 , wherein said plant cells are formulated in a pharmaceutical composition for treating Gaucher's disease comprising the plant cells and a pharmaceutically acceptable carrier. 
     
     
         5 . The method of  claim 4 , wherein said pharmaceutical composition is formulated for oral administration. 
     
     
         6 . A method of increasing hydrolysis of glycosphingolipid glucocerebroside in macrophages of a subject in need thereof, the method comprising administering to said subject a therapeutically effective amount of lyophilized plant cells expressing a recombinant human glucocerebrosidase protein, thereby increasing hydrolysis of glycosphingolipid glucocerebroside in macrophages of the subject. 
     
     
         7 . The method of  claim 6 , wherein said plant cells are carrot cells. 
     
     
         8 . The method of  claim 7 , wherein said carrot cells are from a carrot cell suspension culture. 
     
     
         9 . The method of  claim 6 , wherein said plant cells are formulated in a pharmaceutical composition for treating Gaucher's disease comprising the plant cells and a pharmaceutically acceptable carrier. 
     
     
         10 . The method of  claim 9 , wherein said pharmaceutical composition is formulated for oral administration.