Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II.

ABSTRACT: Mucopolysaccharidosis Type II is a hereditary lysosomal storage disease characterized by deficiency in the enzyme iduronate 2-sulfatase (IDS). IDS is critical in the breakdown of sulfated glycosaminoglycans and its deficiency leads to an accumulation of these compounds across various tissue types resulting in multisystemic dysfunction. Intravenous administration of recombinant IDS (idursulfase) substantially improves patients' quality and length of life. However, recombinant IDS delivered intravenously is sequestered in the liver and respiratory failure remains as the leading cause of death for patients independent of idursulfase treatment, which suggests insufficient delivery to the lungs. This study aimed to assess a novel method of idursulfase administration using a nebulizer in combina

SUBMITTER: Shamoun AJ 

PROVIDER: S-EPMC12200698 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature