庞贝病（也称为糖原贮积病Ⅱ型、酸性麦芽糖酶缺乏症和糖原累积病Ⅱ型）是一种糖原代谢遗传性疾病，由溶酶体酸性α-葡萄糖苷酶(gaa)缺乏引起，该酶可在溶酶体中将糖原降解为葡萄糖。

2015年8月6日 · The key pathway to transport GAA enzyme into the lysosomes in the cell is through the M6P receptor. Nexviazyme is specifically designed to target M6P to improve cellular enzyme uptake and enhance glycogen clearance in target tissues with an approximate 15-fold increase in M6P content compared to alglucosidase alfa, the comparator arm in the ...

2024年1月1日 · In Pompe disease, the CHO-produced recombinant human acid alpha-glucosidase (GAA), alglucosidase alpha, was the first attempt to reach difficult-to-target skeletal muscle and strategies have since been developed to improve the efficacy of the initial enzyme therapy [45]. CI-M6PR addressing is the key to improving therapeutic efficacy and many ...

2016年11月14日 · Herein, we present the synthesis of novel analogues of mannose 6-phosphate (M6P), known as AMFAs and functionalized at the anomeric position for enzyme grafting. AMFAs are non-phosphate serum-resistant derivatives that efficiently bind the cation-independent mannose 6-phosphate receptor (CI-M6PR), which is the main pathway to address enzymes to ...

2018年6月7日 · Chemical conjugation of synthetic M6P glycan was successful in increasing M6P glycan content of rGAA, which enhanced lysosomal targeting and therapeutic efficacy 5,9,10,11,12.

2012年4月1日 · The explanation of how are the lysosomal enzymes accurately recognized and selected over many other proteins in the trans-Golgi network relies on being tagged with a unique marker: the mannose-6-phosphate (M6P) group, which is added exclusively to the N-linked oligosaccharides of lysosomal soluble hydrolases, as they pass through the cis-Golgi ...

To enhance the delivery of rhGAA (recombinant GAA, where GAA stands for acid α-glucosidase) to the affected muscles in Pompe disease, the carbohydrate moieties on the enzyme were remodelled to exhibit a high affinity ligand for the CI-MPR (cation-independent M6P receptor, where M6P stands for mannose 6-phosphate).

2021年1月1日 · As with many other enzymes targeted to the lysosome, endogenous GAA is post-translationally modified with mannose 6-phosphate (M6P), which facilitates high-affinity binding to cation-dependent M6P receptors and cation-independent M6P receptor (CI-MPR), and enables transport to lysosomes from the trans-Golgi network (7, 8, 9).

2005年8月1日 · To enhance the delivery of rhGAA (recombinant GAA, where GAA stands for acid alpha-glucosidase) to the affected muscles in Pompe disease, the carbohydrate moieties on the enzyme were remodelled to exhibit a high affinity ligand for the CI-MPR (cation-independent M6P receptor, where M6P stands for ma …

2023年10月9日 · 艾夫糖苷酶α是第二代酶替代疗法，可以靶向6-磷酸甘露糖（m6p）受体，从而改善gaa向肌肉细胞的递送，使得多余的糖原得以分解，减少对患者肌肉细胞的损伤。