Compositions and methods for treating glycogen storage diseases

Details Compositions and methods for treating glycogen storage diseases Compositions and methods for treating glycogen storage diseases

A - Human Necessities

61

K

A61K 48/00 (2006.01) A61K 38/47 (2006.01) C12N 9/24 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2680063

Compositions including therapeutically effective molecules for treatment of diseases in mammals including glycogen storage diseases (e.g., Pompe disease) and methods of use thereof. These compositions in combination with various routes and methods of administration results in targeted uptake and expression of therapeutic molecules in specific organs, tissues and cells resulting in correction of a disease such as a glycogen storage disease (e.g., Pompe disease).

L'invention concerne des compositions comprenant des molécules thérapeutiques efficaces pour le traitement de maladies chez des mammifères comprenant des maladies liées au stockage du glycogène (par exemple, la maladie de Pompe) et des procédés d'utilisation de celles-ci. Ces compositions en combinaison avec divers voies et procédés d'administration aboutissent à l'absorption ciblée et l'expression de molécules thérapeutiques dans des organes, tissus et cellules spécifiques aboutissant à une correction d'une maladie telle qu'une maladie liée au stockage du glycogène (par exemple, la maladie de Pompe).

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