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Combined Therapies for Lysosomal Storage Diseases

Gabig-Cimińska, Magdalena and Jakóbkiewicz-Banecka, Joanna and Malinowska, Marcelina and Kloska, Anna and Piotrowska, Ewa and Chmielarz, Izabela and Moskot, Marta and Węgrzyn, Alicja and Węgrzyn, Grzegorz (2015) Combined Therapies for Lysosomal Storage Diseases. Current Molecular Medicine, 15 . pp. 746-771.

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Official URL: http://www.eurekaselect.com/135101/article


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.

Item Type:Article
Subjects:Q Science > Q Science (General)
Q Science > QH Natural history
Q Science > QH Natural history > QH301 Biology
Q Science > QH Natural history > QH426 Genetics
Divisions:Laboratory of Molecular Biology (in Gdansk)
ID Code:1134
Deposited By: Prof. Magdalena Gabig-Cimińska
Deposited On:04 Mar 2016 08:54
Last Modified:04 Mar 2016 08:54

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