Neuronal ceroid lipofuscinoses therapeutic strategies: Past, present and future

被引：35
作者：
Hobert, Judith A.
Dawson, Glyn
机构：
[1] Univ Chicago, Dept Pediat, Chicago, IL 60637 USA
[2] Univ Chicago, Comm Mol Metab & Nutr, Div Biol Sci, Chicago, IL 60637 USA
[3] Univ Chicago, Dept Biochem & Mol Biol, Chicago, IL 60637 USA
来源：
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE | 2006年 / 1762卷 / 10期
关键词：
neuronal ceroid lipofuscinoses; chemical chaperone therapy; enzyme therapy; stem cell therapy; gene therapy;
D O I：
10.1016/j.bbadis.2006.08.004
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Historically, many different therapies have been assessed for their ability to alter disease progression of the Neuronal Ceroid Lipofuscinoses (NCLs). While some treatments have lead to minor improvements, none have been able to arrest disease progression or improve the quality or duration of life. Presently, many new therapeutic strategies, such as chaperone therapy, enzyme replacement therapy, gene therapy, and stem cell therapy, are being investigated for their ability to alter the disease course of the NCLs. This review summarizes previous studied therapies,, discusses those currently being evaluated and examines possibilities for future therapies for the treatment of patients with NCL. (c) 2006 Elsevier B.V. All rights reserved.
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收藏
页码：945 / 953
页数：9
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