Synthesis and Evaluation of Bithiazole Derivatives As Potential α-Sarcoglycan Correctors.

ABSTRACT: 4'-Methyl-4,5'-bithiazoles were previously identified as cystic fibrosis transmembrane regulator (CFTR) correctors, thus being able to correct folding defective mutants of the channel regulating chloride transport through the membrane. Additionally, bithiazole derivative C17 was reported to recover α-sarcoglycan in vitro and in vivo. We report here the synthesis of two new derivatives of C17, in which the two sides of the bithiazole scaffold were modified. The synthesized compounds and the corresponding precursors were tested in myogenic cells to evaluate the expression of α-sarcoglycan. The results highlighted that both substitutions of the bithiazole scaffold are important to achieve the maximum recovery of the α-sarcoglycan mutant. Nonetheless, partial preser

SUBMITTER: Ribaudo G 

PROVIDER: S-EPMC10424318 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature