Article

2-Guanidino-quinazoline promotes the readthrough of nonsense mutations underlying human genetic diseases

2021-11-11

Abstract excerpt

Premature termination codons (PTCs) account for 10% to 20% of genetic diseases in humans. The gene inactivation resulting from PTC can be counteracted by the use of drugs stimulating PTC readthrough, thereby restoring production of the full-length protein. However, a greater chemical variety of readthrough inducers is required to broaden the medical applications of this therapeutic strategy. In this study, we deve...

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Literature Corpus work: fce77b93-7a6c-540e-9bce-6723f31d3a07
DOI: 10.1101/2021.11.09.467859

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