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Lysosomal acid alpha-glucosidase (GAA)
- Target
- GAA
- Molecular classification
- Enzyme, Glycosidase
01
Overview
Lysosomal acid alpha-glucosidase (GAA) is a lysosomal enzyme that catalyzes the hydrolysis of α(1→4)-linked glucose residues from glycogen, producing free glucose within lysosomes. It is essential for normal cellular energy metabolism. Deficiency of GAA leads to Pompe disease, characterized by glycogen accumulation in lysosomes. Recombinant human GAA (rhGAA) is used for enzyme replacement therapy. High-resolution crystal structures are available for rhGAA, providing insights into its function and informing drug design.
- Other names
- Acid maltaseGAA
02
Mechanism of action
Enzyme replacement therapy (ERT)
03
Biological functions
Glycogen hydrolysisGlucose productionLysosomal functionEnergy metabolism
04
Disease associations
Pompe disease (Glycogen storage disease type II)
05
Safety considerations
Immune responses to ERTVariable tissue uptake of ERT
06
Interacting drugs
Alglucosidase alfa
1 more in the full profile.
07
Biomarkers
Glycogen levelsEnzyme activity
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