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Current Molecular Medicine


ISSN (Print): 1566-5240
ISSN (Online): 1875-5666

Combined Therapies for Lysosomal Storage Diseases

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

Volume 15, Issue 8, 2015

Page: [746 - 771] Pages: 26

DOI: 10.2174/1566524015666150921105658

Price: $65


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.

Keywords: Combined therapies, enzyme replacement therapy, gene therapy, hematopoietic cell transplantation, lysosomal storage diseases, small molecular chaperones, substrate reduction therapy.

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