Discovery of Novel Chromenopyridine Derivatives as Readthrough-Inducing Drugs
Shota Kawai,
Shunsuke Takashima,
Masafumi Ando
et al.
Abstract:Hurler syndrome, a type of Mucopolysaccharidosis type I, is an inherited disorder caused by the accumulation of glycosaminoglycans (GAG) due to a deficiency in lysosomal α-L-iduronidase (IDUA), resulting in multiorgan dysfunction. In many patients with Hurler syndrome, IDUA proteins are not produced due to nonsense mutations in their genes; therefore, readthrough-inducing compounds, such as gentamycin, are expected to restore IDUA proteins by skipping the premature termination codon. In the present study, we s… Show more
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