IDS Human

Iduronate 2-sulfatase is a glycoprotein with a molecular weight of approximately 61 kDa . It is synthesized as a precursor protein and undergoes post-translational modifications, including glycosylation, to become fully functional . The enzyme’s primary function is to hydrolyze the sulfate ester bond at the 2-position of iduronic acid residues in GAGs .

Recombinant human iduronate 2-sulfatase (rhIDS) is produced using recombinant DNA technology in various host cell systems, such as Chinese hamster ovary (CHO) cells or human cell lines . The recombinant form is designed to mimic the natural enzyme’s structure and function, ensuring its efficacy in therapeutic applications .

The primary therapeutic application of recombinant human iduronate 2-sulfatase is in enzyme replacement therapy (ERT) for patients with Hunter syndrome . The recombinant enzyme is administered intravenously to supplement the deficient or malfunctioning enzyme in affected individuals . This treatment helps reduce the accumulation of GAGs, alleviating symptoms and improving the quality of life for patients .

Idursulfase, marketed under the brand name Elaprase, is a commercially available form of recombinant human iduronate 2-sulfatase . Clinical studies have demonstrated its efficacy in reducing GAG levels in urine and improving various clinical parameters, such as joint mobility, respiratory function, and overall physical endurance . However, the treatment does not cross the blood-brain barrier, limiting its effectiveness in addressing neurological symptoms associated with Hunter syndrome .

Ongoing research aims to improve the delivery and efficacy of recombinant human iduronate 2-sulfatase. Strategies include developing fusion proteins, exploring alternative delivery methods, and investigating gene therapy approaches to provide a more comprehensive treatment for Hunter syndrome .