Ceruloplasmin administration in the preclinical mouse model of aceruloplasminemia reveals a sex-related variation in biodistribution.

• Tác giả: Massimo Alessio, Sara Belloli, Andrea Caricasole, Angela Coliva, Antonio Conti, Cristina Monterisi, Rosa Maria Moresco, Paolo Rainone, Alan Zanardi
• Ngôn ngữ: eng
• Thông tin xuất bản: England : Communications biology , 2025
• Bộ sưu tập: NCBI
• ID: 469298

Tóm tắt

Mutations in the ceruloplasmin (CP) gene are responsible for the rare genetic disease aceruloplasminemia characterized by iron accumulation in different organs, including the brain. We previously reported that administration of purified CP in the CP-deficient (cpKO) mouse model of the disease, was therapeutically effective. Here we evaluated the bioavailability of the therapeutic protein in different organs of the cpKO mouse. The distribution of administered radiolabelled-[