N-acetylmannosamine as potential therapy for Golgi homeostasis disorders

Institution: Department for Children and Adolescents – General Pediatrics, Münster University Hospital
Applicants Dr. Julien H. Park
Funding line:
Else Kröner Memorial Fellowships

Congenital disorders of glycosylation (CDG) are inborn errors of metabolism that lead to severe, potentially fatal multisystem diseases. The project studies how cellular damage occurs due to the lack of sialic acid. The effect of the sialic acid precursor molecule N-acetylmannosamine on sugar chains (glycans) and metabolic processes is being investigated in order to develop a therapy on a long-term basis. Due to the key role of hyposialylation in the case of diseases affecting the central nervous system, the knowledge gained can deepen the understanding of these frequent disorders.

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