420784-05-0
420784-05-0
Chemical Structure
Alglucosidase alfa
Synonym(s): rhGAA
- CAS No.: 420784-05-0
- Formula:N/A
- Molecular Weight:N/A
SMILES: [Alglucosidase alfa]
Biological Activity:
Alglucosidase alfa (rhGAA) is a recombinant human acid α-glucosidase. Alglucosidase alfa is taken up by cells via the cation-independent mannose-6-phosphate receptor (CI-MPR) pathway and transported to lysosomes, where GAA degrades glycogen in the acidic lysosomal environment. Alglucosidase alfa is applicable to research related to lysosomal glycogen metabolism and glycogen storage diseases[1][2][3][4][5].
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Alglucosidase alfa | 98% | Alglucosidase alfa (rhGAA) is a recombinant human acid α-glucosidase. Alglucosidase alfa is taken up by cells via the cation-independent mannose-6-phosphate receptor (CI-MPR) pathway and transported to lysosomes, where GAA degrades glycogen in the acidic lysosomal environment. Alglucosidase alfa is applicable to research related to lysosomal glycogen metabolism and glycogen storage diseases. |
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References
- [1]. Xu S, et al. Improved efficacy of a next-generation ERT in murine Pompe disease. JCI insight. 2019 Mar 07;4(5):e125358.
- [2]. Zafra-Puerta L, et al. Effect of intracerebroventricular administration of alglucosidase alfa in two mouse models of Lafora disease: Relevance for clinical practice. Epilepsy research. 2024 Feb;200:107317.
- [3]. Joly MS, et al. Transient low-dose methotrexate generates B regulatory cells that mediate antigen-specific tolerance to alglucosidase alfa. Journal of immunology (Baltimore, Md. : 1950). 2014 Oct 15;193(8):3947-58. [Content Brief]
- [4]. Yi H, et al. Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV. Molecular genetics and metabolism reports. 2016 Dec;9:31-33. [Content Brief]
- [5]. Dornelles AD, et al. Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis. Frontiers in pediatrics. 2024;12:1310317. [Content Brief]
