Abstract
Genetic code expansion enables in cellulo biosynthesis of curative proteins with enhanced specificity, improved stability, and even novel functions, due to the incorporation of artificial, designed, noncanonical amino acids (ncAAs). In addition, this orthogonal system also holds great potential for in vivo suppressing nonsense mutations during protein translation, providing an alternative strategy for alleviating inherited diseases caused by premature termination codons (PTCs). Here we describe the approach to explore the therapeutic efficacy and long-term safety of this strategy in transgenic mdx mice with stably expanded genetic codes. Theoretically, this method is applicable to about 11% of monogenic diseases involving nonsense mutations.
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