酶替代疗法：概念、乱象与巅峰
Enzyme replacement therapy: conception, chaos and culmination.
作者信息
Brady Roscoe O
机构信息
Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, Building 10, Room 3D04, National Institutes of Health, 9000 Rockville Pike, Bethesda, MD 20892-1260, USA.
出版信息
Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):915-9. doi: 10.1098/rstb.2003.1269.
Soon after the enzymatic defects in Gaucher disease and in Niemann-Pick disease were discovered, enzyme replacement or enzyme supplementation was proposed as specific treatment for patients with these and related metabolic storage disorders. While relatively straightforward in concept, successful implementation of this approach required many years of intensive effort to bring it to fruition. Procedures were eventually developed to produce sufficient quantities of the requisite enzymes for clinical trials and to target therapeutic enzymes to lipid-storing cells. These achievements led to the development of effective enzyme replacement therapy for patients with Gaucher disease and for Fabry disease. These demonstrations provide strong incentive for the application of this strategy for the treatment of many human disorders of metabolism.
在发现戈谢病和尼曼-匹克病的酶缺陷后不久，酶替代或酶补充疗法就被提议作为治疗这些疾病及相关代谢性贮积病患者的特异性治疗方法。虽然这一概念相对简单直接，但要成功实施该方法需要多年的不懈努力才能实现。最终开发出了相应程序，以生产足够数量的用于临床试验的必需酶，并将治疗性酶靶向脂质储存细胞。这些成果促成了针对戈谢病和法布里病患者的有效酶替代疗法的发展。这些实例为将该策略应用于治疗多种人类代谢紊乱疾病提供了强大动力。
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