Journal
ACS MEDICINAL CHEMISTRY LETTERS
Volume 7, Issue 4, Pages 418-423Publisher
AMER CHEMICAL SOC
DOI: 10.1021/acsmedchemlett.6b00006
Keywords
Aminoglycosides; genetic diseases; nonsense mutations; translational readthrough; cystic fibrosis
Categories
Funding
- Eloxx Pharmaceutical LTD Research Fund [2019230]
Ask authors/readers for more resources
New pseudotrisaccharide derivatives of aminoglycosides that exploit additional interaction on the shallow groove face of the decoding-site rRNA of eukaryotic ribosome were designed, synthesized and biologically evaluated. Novel lead structures (6 and 7 with an additional 7'-OH), exhibiting enhanced specificity to eukaryotic cytoplasmic ribosome, and superior nonsense mutation suppression activity than those of gentamicin, were discovered. The comparative benefit of new leads was demonstrated in four different nonsense DNA-constructs underling the genetic diseases cystic fibrosis, Usher syndrome, and Hurler syndrome.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available