Ontology highlight
ABSTRACT:
SUBMITTER: Li S
PROVIDER: S-EPMC10296333 | biostudies-literature | 2023 Jun
REPOSITORIES: biostudies-literature
Li Shan S Li Juan J Shi Wenjing W Nie Ziyan Z Zhang Shasha S Ma Fengdie F Hu Jun J Chen Jianjun J Li Peiqiang P Xie Xiaodong X
Biomolecules 20230614 6
Around 11% of all known gene lesions causing human genetic diseases are nonsense mutations that introduce a premature stop codon (PTC) into the protein-coding gene sequence. Drug-induced PTC readthrough is a promising therapeutic strategy for treating hereditary diseases caused by nonsense mutations. To date, it has been found that more than 50 small-molecular compounds can promote PTC readthrough, known as translational readthrough-inducing drugs (TRIDs), and can be divided into two major categ ...[more]