摘要
溶酶体贮积病(LSDs)包括一组罕见的能引起溶酶体内特定天然酶不足的遗传慢性综合症。那些常被溶酶体酶代谢分解的大分子化合物积累在这些细胞器内,造成对组织,骨骼和器官的渐进破坏,在一些情况下,还会破坏中枢神经系统(CNS)。这些破坏会导致基板累积最后造成体质下降,功能障碍和潜在的死亡。到目前为止,最确定的LSDs治疗方法是酶替代疗法,它给病人提供了相应的活性酶。然而,这些酶在血液中没有足够稳定性,因此治疗必须在医生的指导下进行周期性的静脉注射,并且免疫原性反应频现。此外,中枢神经系统所在的区域,血脑屏障(BBB)是一个主要的障碍,酶不能到达。纳米技术可以提供有用的障碍来成功保护和保存酶,并运输他们穿过血脑屏障到达脑部。一些基于定向BBB特殊受体的战略,使纳米粒子成功携带敏感分子到达脑部。然后,此文描述了主要的溶酶体贮积病,并提供了一个最新脑内分布纳米战略的详细综述。
关键词: 溶酶体贮积病,高雪氏病,酶替代疗法,纳米技术,定向
Current Medicinal Chemistry
Title:Role of Nanotechnology for Enzyme Replacement Therapy in Lysosomal Diseases. A Focus on Gaucher’s Disease
Volume: 23 Issue: 9
Author(s): L. Martín-Banderas, M.A. Holgado, M. Durán-Lobato, J.J. Infante, J. Álvarez-Fuentes, M. Fernández-Arévalo
Affiliation:
关键词: 溶酶体贮积病,高雪氏病,酶替代疗法,纳米技术,定向
摘要: Lysosomal storage diseases (LSDs) comprise a group of rare inherited chronic syndromes that cause deficiency of specific native enzymes within the lysosomes. The macromolecular compounds that are usually catabolized by lysosomal enzymes are accumulated within these organelles, causing progressive damage to tissues, skeleton and organs and, in several cases, the central nervous system (CNS). The damage caused by substrate accumulation finally results in physical deterioration, functional impairment and potential death. Up to date, the most promising therapy for most LSDs is enzyme-replacement therapy (ERT), which provides patients with the corresponding active enzyme. However, these enzymes do not have enough stability in blood, the treatment must be therefore periodically administrated by i.v. infusion under medical supervision, and immunogenicity issues are frequent. In addition, affected areas within the CNS, where the blood-brain barrier (BBB) is a major obstacle, cannot be reached by the enzymes. Nanotechnology can provide useful carriers to successfully protect and preserve enzymes, and transport them through the BBB towards brain locations. Several strategies based on targeting specific receptors on the BBB have led to nanoparticles that successfully carry sensitive molecules to the brain. Then, the main LSDs are described and a thorough review of nanotechnology strategies for brain delivery studied up to date is presented.
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Cite this article as:
L. Martín-Banderas, M.A. Holgado, M. Durán-Lobato, J.J. Infante, J. Álvarez-Fuentes, M. Fernández-Arévalo , Role of Nanotechnology for Enzyme Replacement Therapy in Lysosomal Diseases. A Focus on Gaucher’s Disease, Current Medicinal Chemistry 2016; 23 (9) . https://dx.doi.org/10.2174/0929867323666160210130608
DOI https://dx.doi.org/10.2174/0929867323666160210130608 |
Print ISSN 0929-8673 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-533X |
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