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脂质贮积病的酶替代疗法研究。

Investigations in enzyme replacement therapy in lipid storage diseases.

作者信息

Brady R O, Pentchev P G, Gal A G

出版信息

Fed Proc. 1975 Apr;34(5):1310-5.

PMID:804420
Abstract

Enzyme replacement appears to offer much promise as specific therapeutic procedures for patients with Fabry's disease and Gaucher's disease. However, enzyme replacement in patients with Tay-Sachs disease and other heritable metabolic disorders where the central nervous system is affected will require first the development of effective methods for the delivery of exogenous enzymes to the brain; such methods are not yet available.

摘要

酶替代疗法对于法布里病和戈谢病患者而言,似乎有望成为特定的治疗手段。然而,对于中枢神经系统受影响的泰-萨克斯病及其他遗传性代谢紊乱患者,酶替代疗法首先需要开发出将外源性酶输送至大脑的有效方法;目前尚无此类方法。

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