伴侣蛋白病与伴侣蛋白疗法。

Chaperonopathies and chaperonotherapy.

作者信息

Macario Alberto J L, Conway de Macario Everly

机构信息

University of Maryland Biotechnology Institute (UMBI), Columbus Center, 701 E. Pratt Street, Baltimore, MD 21202, USA.

出版信息

FEBS Lett. 2007 Jul 31;581(19):3681-8. doi: 10.1016/j.febslet.2007.04.030. Epub 2007 Apr 24.

DOI:10.1016/j.febslet.2007.04.030

PMID:17475257

Abstract

The study of molecular chaperones (genetics, structure, location, physiology, pathology, and therapeutics) has developed into a science with specific objectives, methods, and hypotheses, a discipline we called chaperonology. Subdisciplines of chaperonology include the study of pathological chaperones (chaperonopathies) and the analysis of their genes in sequenced genomes (chaperonomics). Chaperonopathies are pathological conditions in which one type of chaperone is deficient due to a genetic or acquired defect that modifies the chaperone's structure and/or makes the chaperone unavailable for functioning when needed. Experimental and clinical data show that chaperones and their genes can be used for treating various pathological conditions, thus justifying the development of chaperonotherapy. We discuss recent work showing that chaperonotherapy is on solid foundations: the data demonstrate that molecular chaperones counteract pathogenetic mechanisms in disease and during stress.

摘要

分子伴侣（遗传学、结构、定位、生理学、病理学及治疗学）的研究已发展成为一门具有特定目标、方法和假设的科学，我们将这一学科称为伴侣蛋白学。伴侣蛋白学的子学科包括病理性伴侣蛋白（伴侣蛋白病）的研究以及在已测序基因组中对其基因的分析（伴侣蛋白组学）。伴侣蛋白病是一类病理状态，其中某一种伴侣蛋白因遗传或后天缺陷而缺乏，这种缺陷会改变伴侣蛋白的结构和/或使其在需要时无法发挥功能。实验和临床数据表明，伴侣蛋白及其基因可用于治疗各种病理状态，从而为伴侣蛋白疗法的发展提供了依据。我们讨论了近期的研究工作，这些工作表明伴侣蛋白疗法有着坚实的基础：数据证明分子伴侣可对抗疾病和应激过程中的致病机制。