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.

Conflict of Interest

The authors declared no conflicts of interest with respect to the authorship and/or publication of this article.

Financial Disclosure

The authors received no financial support for the research and/or authorship of this article.