Acid alpha-glucosidase and fragments thereof

Details Acid alpha-glucosidase and fragments thereof Acid alpha-glucosidase and fragments thereof

C - Chemistry, Metallurgy

12

N

C12N 9/24 (2006.01) A61K 38/47 (2006.01) C12N 15/62 (2006.01)

Patent

CA 2553955

Targeted acid alpha-glucosidase therapeutics that localize to the lysosome are provided. The targeted therapeutics include a therapeutic agent, GAA, and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.

Cette invention concerne des agents thérapeutiques ciblés à base d'alpha-glucosidase acide qui sont localisés au niveau du lysosome. Ces agents thérapeutiques ciblés comprennent un agent thérapeutique, l'alpha-glucosidase acide, et une fraction de ciblage qui se lie à un récepteur sur une surface extérieure de la cellule, ce qui permet d'obtenir une localisation infracellulaire correcte de l'agent thérapeutique ciblé dès l'internalisation du récepteur. Cette invention concerne également des acides nucléiques, des cellules et des méthodes associés à la mise en oeuvre de cette invention.

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