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Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease mice

机译：靶向递送酸性α-葡糖苷酶校正Pompe疾病小鼠中的骨骼肌表型

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Andrew D. Baik; Philip T. Calafati; Nina A. Aaron; Antonia Mehra; Sven Moller-Tank; Lawrence Miloscio; Lili Wang; Maria Praggastis; Matthew S. Birnbaum; Cheryl Pan; Susannah Brydges; Alejandro Mujica; Peter Barbounis; Nicholas W. Gale; Ning Li; Christos A. Kyratsous; Christopher J. Schoenherr; Andrew J. Murphy; Aris N. Economides; Katherine D. Cygnar;
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1. Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme. [J] . Raben N, Jatkar T, Lee A, Molecular therapy: the journal of the American Society of Gene Therapy . 2002,第5期

机译：酸性α-葡萄糖苷酶突变体（Pompe）小鼠的骨骼肌中储存的糖原对心肌的抵抗力很高，而糖原对心肌中的糖原的抵抗力很高。
2. Carbohydrate-remodel led acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice [J] . Zhu YX, Li XM, McVie-Wylie A, The Biochemical Journal . 2005,第3期

机译：对阳离子非依赖性甘露糖6磷酸受体具有更高亲和力的碳水化合物重塑的铅酸α-葡萄糖苷酶显示出对庞贝小鼠肌肉的改善递送
3. Histological examination of the effect of a highly phosphorylated proprietary recombinant human acid alpha-glucosidase on glycogen reduction in disease-relevant muscles of Pompe mice [J] . Lun Yi, Xu Su, Gotschall Russell, Molecular genetics and metabolism . 2015,第2期

机译：组织学检查高度磷酸化的专有重组人酸性α-葡萄糖苷酶对庞贝小鼠疾病相关肌肉糖原减少的影响
4. Effect of a Degradable Methacrylic Acid-Based Hydrogel on Macrophage Phenotype in Skeletal Muscle [C] . Miranda M. Carleton, Michael V. Sefton Society for Biomaterials annual meeting and exposition . 2019

机译：可降解的基于甲基丙烯酸的水凝胶对骨骼肌巨噬细胞表型的影响
5. Utilization of Lyso-Phosphatidylserine Nanoparticles to Reduce Immunogenicity and Improve Clinical Utility of Acid Alpha-Glucosidase in the Treatment of Pompe Disease [D] . Dingman, Robert Kenneth. 2020

机译：利用氯磷酰胺纳米粒子以减少酸α-葡糖苷酶治疗Pompe病的免疫原性和改善酸α-葡糖苷酶的临床用途
6. High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease. [O] . J L Van Hove, H W Yang, J Y Wu, 1996

机译：在中国仓鼠卵巢细胞中大量生产重组人溶酶体酸α-葡萄糖苷酶其靶向心肌并纠正庞贝病患者成纤维细胞中糖原的积累。
7. High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease. [O] . Van Hove, J L, Yang, H W, Wu, J Y, 1996

机译：在中国仓鼠卵巢细胞中大量生产重组人溶酶体酸α-葡萄糖苷酶，其靶向心肌并纠正庞贝病患者成纤维细胞中糖原的积累。

Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease mice

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