Afegostat TFA is a pharmacological chaperone that selectively and reversibly binds to acid-β-glucosidase (GCase) within the endoplasmic reticulum. This interaction enhances the stability and activity of GCase, addressing enzyme deficiencies associated with Gaucher disease. Afegostat TFA is utilized in research exploring enzyme therapy and cellular mechanisms of lysosomal storage disorders.
Afegostat TFA is a pharmacological chaperone that selectively and reversibly binds to acid-β-glucosidase (GCase) within the endoplasmic reticulum. This interaction enhances the stability and activity of GCase, addressing enzyme deficiencies associated with Gaucher disease. Afegostat TFA is utilized in research exploring enzyme therapy and cellular mechanisms of lysosomal storage disorders.
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