Methods and materials for treatment of pompe's disease

An amino acid, molecular complex technology, applied in chemical instruments and methods, biochemical equipment and methods, pharmaceutical formulations, etc., can solve problems such as expensive
CN104379162AActive Publication Date: 2015-02-25OXYRANE UK
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Patent Information

Authority / Receiving Office
CN · China
Current Assignee / Owner
OXYRANE UK
Publication Date
2015-02-25

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Abstract

This document relates to molecular complexes having acid alpha glucosidase activity and at least one modification that results in enhanced ability of the molecular complex to be transported to the interior of a mammalian cell.
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Description

[0001] Cross References to Related Applications

[0002] This application claims the benefit of US Provisional Application Serial No. 61 / 611,485, filed March 15, 2012. The disclosure of the prior application is considered part of (and is incorporated by reference into) the disclosure of the present application. field of invention

[0003] The present invention relates to isolated molecular complexes having acid alpha glucosidase activity, and more particularly to molecular complexes comprising at least two polypeptides derived from precursor molecules by proteolysis, wherein the molecular complexes comprise at least one A modification that results in an increased ability of the molecular complex to be transported inside the mammalian cell. Background of the invention

[0004] Pompe's disease (also known as glycogen-storage disease type II or acid maltase deficiency) is a rare autosomal recessive disorder in which acid alpha glucosidase (GAA ) deficiency leads to the accumu...

Claims

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