For diseases caused by nonsense mutations. By using a viral vector to express a patented tRNA in targeted cells, we insert the normal amino acid at the site of the premature stop codon, generating a normal functional protein. Because many diseases share same premature stop codon, the same Suppressor tRNA can treat multiple diseases.
For diseases caused by haploinsufficiency. By using a viral vector to overexpress a unique combination of endogenous tRNAs in targeted cells, we increase the amount of protein produced by the wild-type (functional) copy of the specific gene to its normal level by increasing the stability and thus the half-life of its mRNA.