Birmingham Women's and Children's Hospital, NHS Foundation Trust, Birmingham B4 6NH, UK.
Nutrients. 2021 Feb 2;13(2):484. doi: 10.3390/nu13020484.
Protein substitutes developed for phenylketonuria (PKU) are a synthetic source of protein commonly based on L-amino acids. They are essential in the treatment of phenylketonuria (PKU) and other amino acid disorders, allowing the antagonistic amino acid to be removed but with the safe provision of all other amino acids necessary for maintaining normal physiological function. They were first formulated by a chemist and used experimentally on a 2-year-old girl with PKU and their nutritional formulations and design have improved over time. Since 2008, a bioactive macropeptide has been used as a base for protein substitutes in PKU, with potential benefits of improved bone and gut health, nitrogen retention, and blood phenylalanine control. In 2018, animal studies showed that physiomimic technology coating the amino acids with a polymer allows a slow release of amino acids with an improved physiological profile. History has shown that in PKU, the protein substitute's efficacy is determined by its nutritional profile, amino acid composition, dose, timing, distribution, and an adequate energy intake. Protein substitutes are often given little importance, yet their pharmacological actions and clinical benefit are pivotal when managing PKU.
为苯丙酮尿症（PKU）开发的蛋白质替代品是一种常用的合成蛋白质来源，通常基于 L-氨基酸。它们在治疗苯丙酮尿症（PKU）和其他氨基酸代谢紊乱方面必不可少，既能去除拮抗氨基酸，又能安全提供维持正常生理功能所需的所有其他氨基酸。这些替代品最初是由一位化学家开发的，并在一名 2 岁的 PKU 女孩身上进行了实验性应用，其营养配方和设计随着时间的推移而不断改进。自 2008 年以来，一种生物活性大肽已被用作 PKU 蛋白质替代品的基础，具有改善骨骼和肠道健康、氮潴留和血液苯丙氨酸控制的潜在益处。2018 年，动物研究表明，用聚合物包裹氨基酸的仿生技术可实现氨基酸的缓慢释放，并改善其生理特性。历史表明，在 PKU 中，蛋白质替代品的疗效取决于其营养状况、氨基酸组成、剂量、时间、分布和足够的能量摄入。蛋白质替代品往往被忽视，但在管理 PKU 时，其药理作用和临床益处至关重要。
