Augmentation Therapy with Alpha-1 Antitrypsin: Present and Future
of Production, Formulation, and Delivery

doi:10.2174/0929867328666210525161942
摘要

α1-抗胰蛋白酶是30多年前市场上推出的首批蛋白质疗法之一，迄今为止，它仅用于治疗称为α-1抗胰蛋白酶缺乏症的遗传病的严重形式。唯一批准的制剂来自等离子体，这带来了与其供应有限和高处理成本相关的潜在问题。此外，使用α-1抗胰蛋白酶的增强治疗仍然仅限于静脉输注，这对患者来说是一种繁琐的方案。在这里，我们回顾了最近关于其未来可能发展的文献，重点是i）血浆衍生蛋白的重组替代品，ii）新制剂和iii）新型给药途径。α-1抗胰蛋白酶的监管问题和仍然不清楚的非规范功能，可能与仅在血浆衍生蛋白中发现的糖基化模式有关，阻碍了新产品的引入。然而，除了治疗α-1抗胰蛋白酶缺乏症之外，潜在的新治疗适应症可能会为新的来源和新配方开辟道路。
关键词: α-抗胰蛋白酶，α-蛋白酶抑制剂，中性粒细胞弹性蛋白酶，增强疗法，蛋白质治疗制剂，肺部药物递送。
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DOI https://dx.doi.org/10.2174/0929867328666210525161942	Print ISSN 0929-8673
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当代药物化学

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