BiochemistryNucleic Acids ResearchPeer-reviewed
Mechanism-based approach in designing patient-specific combination therapies for nonsense mutation diseases
Saleem Y. Bhat, A.A. Bhattacharya, Hong Li…·Barry S. Cooperman
This paper investigates how PTC identity and sequence context affect readthrough efficiency and ataluren's effectiveness, providing insights into patient-specific therapy design.
7 citations