Lysosomal targeting pathway enzymes

Details Lysosomal targeting pathway enzymes Lysosomal targeting pathway enzymes

C - Chemistry, Metallurgy

12

N

C12N 15/54 (2006.01) A61K 38/46 (2006.01) C07H 21/04 (2006.01) C07K 14/00 (2006.01) C12N 1/20 (2006.01) C12N 5/10 (2006.01) C12N 9/12 (2006.01) C12N 9/14 (2006.01) C12N 9/16 (2006.01) C12N 15/00 (2006.01) C12N 15/63 (2006.01) C12P 21/00 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2383217

The lysosomal targeting pathway enzymes GlcNAc-phosphotransferase and phosphodiester .alpha.-GlcNAcase are recovered and used to produce highly phosphorylated lysosomal hydrolases that can be used to treat lysosomal storage diseases. Generally, the nucleic acid molecules coding for the enzymes are incorporated into expression vectors that are used to transfect host cells that express the enzymes. The expressed enzymes are recovered and used to treat lysosomal hydrolases having high mannose structures. The resulting lysosomal hydrolases have asparagine-linked oligosaccharides that are highly phosphorylated with mannose phosphate. The highly phosphorylated lysosomal hydrolases are readily taken into the cell and into the lysosome during enzyme replacement therapy procedures.

L'invention concerne des enzymes GlcNac-phosphotransférase et phosphodiester .alpha.-GlcNAcase à voie de ciblage lysosomial, qui sont récupérées et utilisées pour produire des hydrolases lysosomiales fortement phosphorylées pouvant servir à traiter les maladies lysosomales. Les molécules d'acide nucléique codant pour les enzymes sont généralement incorporées dans des vecteurs d'expression qui sont utilisés pour transfecter des cellules hôtes qui expriment les enzymes. Les enzymes exprimées sont récupérées et utilisées pour traiter les hydrolases lysosomiales ayant des structures de mannose élevées. Les hydrolases lysosomiales résultantes comportent des oligosaccharides liés à l'asparagine qui sont fortement phosphorylés avec du mannose-phosphate. Les hydrolases lysosomiales fortement phosphorylées sont rapidement absorbées dans la cellule et dans le lysosome au cours de thérapies de substitution.

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