Document Type
Article
Publication Date
7-7-2018
Abstract
Despite many beneficial outcomes of the conventional enzyme replacement therapy (ERT), several limitations such as the high-cost of the treatment and various inadvertent side effects including the occurrence of an immunological response against the infused enzyme and development of resistance to enzymes persist. These issues may limit the desired therapeutic outcomes of a majority of the lysosomal storage diseases (LSDs). Furthermore, the biodistribution of the recombinant enzymes into the target cells within the central nervous system (CNS), bone, cartilage, cornea, and heart still remain unresolved. All these shortcomings necessitate the development of more effective diagnosis and treatment modalities against LSDs. Taken all, maximizing the therapeutic response with minimal undesired side effects might be attainable by the development of targeted enzyme delivery systems (EDSs) as a promising alternative to the LSDs treatments, including different types of mucopolysaccharidoses (MPSs ) as well as Fabry, Krabbe, Gaucher and Pompe diseases.
Recommended Citation
Safary, Azam; Khiavi, Mostafa Akbarzadeh; Mousavi, Rahimeh; Barar, Jaleh; and Rafi, Mohammad, "Enzyme replacement therapies: What is the best option?" (2018). Department of Neurology Faculty Papers. Paper 162.
https://jdc.jefferson.edu/neurologyfp/162
Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial 4.0 License
Language
English
Comments
This article has been peer reviewed. It is the author’s final published version in BioImpacts, Volume 8, Issue 3, July 2018, Pages 153-157.
The published version is available at https://doi.org/10.15171/bi.2018.17. Copyright © Safary et al.