Over the past three decades, enzyme therapy for lysosomal storage diseases has moved from an academic pursuit to direct delivery of effective clinical care for affected patients and families. This success is based on understanding the complexities of lysosomal biogenesis, lysosomal hydrolase sorting and hydrolytic requirements, and the target sites of pathology of these diseases. This article reviews these concepts and their application to the treatment of affected patients with Gaucher disease, Fabry disease, and mucopolysaccharidosis I. The principles, progress, and practice in these diseases provide prototypes for expansion of enzyme therapy to a growing set of these diseases.

中文翻译：

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溶酶体贮积病的酶疗法：原理，实践和前景。

在过去的三十年中，用于溶酶体贮积病的酶治疗已从学术追求转向直接为受影响的患者和家庭提供有效的临床护理。成功的基础是对溶酶体生物发生的复杂性，溶酶体水解酶的分类和水解要求以及这些疾病的病理学靶点的了解。本文回顾了这些概念及其在治疗戈谢病，法布里病和粘多糖贮积病I的患者中的应用。这些疾病的原理，进展和实践为将酶治疗扩展到越来越多的此类疾病提供了原型。