IDUA抗体;IDUA Antibody—艾普蒂

IDUA antibodies target the enzyme alpha-L-iduronidase (IDUA), a lysosomal hydrolase encoded by the *IDUA* gene. This enzyme catalyzes the breakdown of glycosaminoglycans (GAGs), particularly dermatan and heparan sulfate. Mutations in *IDUA* cause mucopolysaccharidosis type I (MPS I), a lysosomal storage disorder characterized by GAG accumulation, leading to multisystemic symptoms ranging from severe (Hurler syndrome) to attenuated (Scheie syndrome) forms. IDUA antibodies are primarily studied in two contexts. First, they are used as research tools to detect IDUA protein levels in diagnostic assays or to study enzyme function and localization in cellular models. Second, in therapeutic settings, anti-IDUA antibodies may emerge as neutralizing agents in patients receiving enzyme replacement therapy (ERT), such as laronidase (recombinant IDUA), potentially reducing treatment efficacy. Monitoring antibody responses is critical for managing ERT outcomes. Additionally, IDUA antibodies are explored in gene therapy research to assess immune responses against the enzyme post-vector administration. Understanding IDUA antibody dynamics helps optimize therapeutic strategies for MPS I and informs the development of novel treatments, including immunomodulatory approaches to mitigate antibody-mediated inhibition. Their role in both diagnostics and therapeutics underscores their importance in advancing MPS I research and patient care.