Between 10 to 15% of all genetic diseases have the same underlying cause: a nonsense mutation. 1 This type of mutation results from a stop codon in the middle of a gene sequence, which terminates ...

(A) Suppressor tRNA readthrough strategy. Natural tRNAs with engineered anticodons are charged with canonical amino acids to enable readthrough of the premature termination codon (PTC). (B) Unnatural ...

Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without ...

This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...