ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS?

Abstract

Lysosomal storage disorders are an agglomeration of genetic disorders such as Fabry disease, Gaucher disease, Pompe disease, Krabbe’s disease and mucopolysaccharidosis that typically impairs the prime orangs of humans, including brain, heart, musculoskeletal system, spleen, eye, and lungs. Patients with lysosomal storage disorders face mild to severe complications and even death. In order to address these health concerns, scientists are working by dint off, various therapies are introduced such as gene therapy, typical oral medicines, organ/ cell transplantation etc. However, hematopoietic stem cell transplantation and enzyme replacement therapy came out as best stakeholders to treat aforementioned disorders. Nonetheless, according to suggested data, it is concluded that presently enzyme replacement therapies are somehow ineffective for many lysosomal storage disorders till today. But we believe that in near future, as more and more research will be progressed, the ultimate therapy to these disorders will be developed.

Keywords

• drug delivery systems
• enzyme replacement therapy
• fabry disease
• lysosomal enzyme disorders
• lysosomal storage disorders
• gaucher disease
• pompe disease.