A new type of drug, PTC 124, enables translation to continue through the nonsense codon. Trials in mice homozygous for a CFTR allele containing the nonsense codon have found that animals treated with PTC 124 produce normal CFTR protein in their cells. The drug is taken orally and is readily taken up into cells all over the body.
Using your knowledge of the progress towards successful gene therapy for cystic fibrosis, suggest why PTC 124 could be simpler and more reliable treatment for this disease.