paromamine and Mucopolysaccharidosis-I

paromamine has been researched along with Mucopolysaccharidosis-I* in 1 studies

Other Studies

1 other study(ies) available for paromamine and Mucopolysaccharidosis-I

ArticleYear
Repairing faulty genes by aminoglycosides: development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations.
    Bioorganic & medicinal chemistry, 2010, Jun-01, Volume: 18, Issue:11

    New pseudo-di- and pseudo-trisaccharide derivatives of the aminoglycoside drug G418 were designed, synthesized and their ability to readthrough nonsense mutations was examined in both in vitro and ex vivo systems, along with the toxicity tests. Two novel lead structures, NB74 and NB84, exhibiting significantly reduced cell toxicity and superior readthrough efficiency than those of gentamicin, were discovered. The superiority of new leads was demonstrated in six different nonsense DNA-constructs underling the genetic diseases cystic fibrosis, Duchenne muscular dystrophy, Usher syndrome and Hurler syndrome.

    Topics: Aminoglycosides; Animals; Codon, Nonsense; Cystic Fibrosis; Drug Design; Genetic Diseases, Inborn; Genetic Techniques; Gentamicins; Humans; Mucopolysaccharidosis I; Muscular Dystrophy, Duchenne; Trisaccharides; Usher Syndromes

2010