Mucopolysaccharidosis and Therapeutic Agents
Selime Özakyol1, Mine Ceren Akbulut1, Oytun Erbaş1
1ERBAS Institute of Experimental Medicine, Illinois, USA & Gebze, Türkiye
Keywords: Enzyme replacement therapy, hematopoietic stem cell transplantation, mucopolysaccharidosis, therapeutic agent
Abstract
Mucopolysaccharidosis (MPS) is a lysosomal storage disease that causes the accumulation of glycosaminoglycans (GAGs) in cells and occurs with the failure of hydrolysis enzymes to function, which divides big molecules into small molecules. Accumulation of GAGs in cells, tissues, and organs causes vital problems. Although various treatments, such as enzyme replacement or hematopoietic stem cell therapy, have been applied, a definitive treatment has yet to be found. This review discusses MPS and its treatment options.
Cite this article as: Özakyol S, Akbulut MC, Erbaş O. Mucopolysaccharidosis and Therapeutic Agents. JEB Med Sci 2022;3(3):199-205.
The authors declared no conflicts of interest with respect to the authorship and/or publication of this article.
The authors received no financial support for the research and/or authorship of this article.