Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses

被引：1
作者：
Pierret, Chris [1 ]
Morrison, Jason A. [1 ]
Kirk, Mark D. [1 ]
机构：
[1] Univ Missouri, Div Biol Sci, Columbia, MO 65211 USA
来源：
ACTA NEUROBIOLOGIAE EXPERIMENTALIS | 2008年 / 68卷 / 03期
关键词：
lusosomal storage; neuronal ceroid-lipofuscinoses; enzyme replacement; stem cell; gene therapy; substrate reduction;
D O I：
暂无
中图分类号：
Q189 [神经科学];
学科分类号：
071006 ;
摘要：
Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments arc considered across the LSDs and extended to therapies for the NCLs. It is likely that I combination of approaches will produce the most beneficial clinical outcome for treatment of pathologies displayed by the NCLs.
引用
收藏
页码：429 / 442
页数：14
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