| 摘要 |
<p>The present invention relates to compositions and methods for treating glycogen storage disease, such as GSD II. A human activator enzyme, termed ASA, of acid α a-glycosidase, (GAA) or acid maltase is specifically defined and characterized. The AGA has been found to increase the activity of human acid α glycosidase activity by at least 10-fold, relative to the activity of GAA in the absence of the activator protein. The invention thus also provides for a method of increasing the activity of GAA, particularly through the action of AGA. The AGA has an approximate molecular weight of 25-30 kD, and is found to be heat stable. In addition, the AGA is found to have an extended shelf-life without significant loss of ability to activate GAA.</p> |
