自噬，分子伴侣，内质网应激，细胞凋亡，未折叠蛋白反应。

Title:Combined Therapies for Lysosomal Storage Diseases

Volume: 15 Issue: 8

Author(s): M. Gabig-Cimińska, J. Jakóbkiewicz-Banecka, M. Malinowska, A. Kloska, E. Piotrowska and I. Chmielarz, M. Moskot, A. Węgrzyn and G. Węgrzyn

Affiliation:

关键词: 联合疗法，酶替代疗法，基因治疗，造血干细胞移植，溶酶体贮积病，小分子伴侣，减少底物治疗。

摘要: Lysosomal storage diseases (LSDs) is a group consisting of over 50 disorders caused mostly by dysfunctions of lysosomal proteins and resultant accumulation of particular compounds inside cells and extracellular volumes in affected organisms. Genetic diseases are among the most difficult targets for medical treatment. Nevertheless, understanding of molecular bases of LSDs made it possible to develop novel procedures of treatment, employing molecular medicine. Although various therapeutic approaches have been proposed, and some of them were introduced into clinical practice, none of them was found to be effective in correcting all symptoms in treated patients. Central nervous system and skeleton appear to be the most difficult targets to be improved. Therefore, a proposal appeared that perhaps no single therapeutic procedure may be fully effective in treatment of LSD patients, and only combination of two or more approaches could be a successful therapy. In this review, we present and discuss current stage of various combination therapies for LSDs, based on already available published data.

Cite this article as:

M. Gabig-Cimińska, J. Jakóbkiewicz-Banecka, M. Malinowska, A. Kloska, E. Piotrowska and I. Chmielarz, M. Moskot, A. Węgrzyn and G. Węgrzyn , Combined Therapies for Lysosomal Storage Diseases, Current Molecular Medicine 2015; 15 (8) . https://dx.doi.org/10.2174/1566524015666150921105658