Murine Fibroblasts and Primary Hepatocytes as Tools When Studying the Efficacy of Potential Therapies for Mucopolysaccharidosis Type I.

ABSTRACT: Mucopolysaccharidosis type I (MPS I) is a metabolic genetic disease caused by the deficiency of a lysosomal enzyme involved in glycosaminoglycans (GAGs) degradation. MPS I cells have a constant level of GAG synthesis, but disturbed degradation means that GAGs accumulate progressively, impairing cell metabolism. GAG metabolism can be modulated by flavonoids, and these are being studied as therapeutics for MPS. We have optimised the protocol for obtaining fibroblasts and hepatocytes from the MPS I murine model and characterised the cells for their suitability as an in vitro model for testing compounds with therapeutic potential. Methods: Murine primary hepatocytes and fibroblasts were used as a cellular model to study the effect of genistein, biochanin A, and kaempferol on the modulation of

SUBMITTER: Wesierska M 

PROVIDER: S-EPMC9820816 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature