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Claims for Patent: 8,227,230

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Claims for Patent: 8,227,230

Title:Human lysosomal proteins from plant cell culture
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.
Inventor(s): Shaaltiel; Yoseph (Kibbutz HaSolelim-Doar-Na HaMovil, IL), Baum; Gideon (Kibbutz Ayelet HaShachar-D.N. Upper Galilee, IL), Bartfeld; Daniel (Moran--Doar-Na Bikat Beit HaKerem, IL), Hashmueli; Sharon (Ramot-Naftali, IL), Lewkowicz; Ayala (Kfar-Vradim, IL)
Assignee: Protalix Ltd. (Carmiel, IL)
Application Number:13/080,692
Patent Claims: 1. A recombinant human glucocerebrosidase protein comprising an amino acid sequence as set forth in SEQ ID NO: 15.

2. The recombinant human glucocerebrosidase protein of claim 1, being glycosylated with at least one exposed mannose residue.

3. The recombinant human glucocerebrosidase protein of claim 1, further comprising at least one fucose residue having an alpha (1-3) glycosidic bond.

4. The recombinant human glucocerebrosidase protein of claim 1, further comprising at least one core xylose residue.

5. The recombinant human glucocerebrosidase protein of claim 2, being glycosylated with at least one core xylose residue and at least one fucose residue having an alpha (1-3) glycosidic bond.

6. The recombinant human glucocerebrosidase protein of claim 2, wherein said glucocerebrosidase protein binds to a mannose receptor on a macrophage.

7. The recombinant human glucocerebrosidase protein of claim 6, having an increased uptake in said macrophages, in comparison with the corresponding uptake of a mammalian cell-expressed glucocerebrosidase protein by said macrophages.

8. A pharmaceutical composition for treating Gaucher's disease comprising the recombinant human glucocerebrosidase protein of claim 1 and a pharmaceutically acceptable carrier, said pharmaceutical composition formulated for parenteral administration.
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