Jeyakumar Mylvaganam, Dwek Raymond A, Butters Terry D, Platt Frances M
Department of Biochemistry, University of Oxford, UK.
Nat Rev Neurosci. 2005 Sep;6(9):713-25. doi: 10.1038/nrn1725.
Many neurodegenerative diseases are characterized by the accumulation of undegradable molecules in cells or at extracellular sites in the brain. One such family of diseases is the lysosomal storage disorders, which result from defects in various aspects of lysosomal function. Until recently, there was little prospect of treating storage diseases involving the CNS. However, recent progress has been made in understanding these conditions and in translating the findings into experimental therapies. We review the developments in this field and discuss the similarities in pathological features between these diseases and some more common neurodegenerative disorders.
许多神经退行性疾病的特征是细胞内或大脑细胞外部位存在不可降解分子的积累。溶酶体贮积症就是这样一类疾病，它是由溶酶体功能各个方面的缺陷引起的。直到最近，治疗涉及中枢神经系统的贮积症的前景还很渺茫。然而，在理解这些病症以及将研究结果转化为实验性疗法方面，最近已经取得了进展。我们回顾了该领域的进展，并讨论了这些疾病与一些更常见的神经退行性疾病在病理特征上的相似之处。
