Article

The use of genistein and ambroxol is an effective approach in correcting cellular dysfunctions of mucopolysaccharidosis-plus syndrome

2025-09-04

Abstract excerpt

<title>Abstract</title> <p> Mucopolysaccharidosis-plus syndrome (MPS plus or MPSPS) is an ultrarare inherited metabolic disease, caused by mutations in the <italic>VPS33A</italic> gene. Like in different types of mucopolysaccharidosis (MPS), glycosaminoglycan (GAG) storage in cells of patients is evident. However, unlike MPS, the genetic defects in MPSPS cause impairment in the VPS33A protein level rather than...

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Literature Corpus work: 74db2b45-817e-5179-897b-bba1d69f5d33
DOI: 10.21203/rs.3.rs-7429141/v1

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