NUCLEIC ACID SEQUENCE AND METHOD FOR SELECTIVELY EXPRESSING A PROTEIN IN A TARGET CELL OR TISSUE

摘要

A synthetic nucleic acid sequence and a method are disclosed for selectively expressing a protein in a target cell or tissue of a mammal. Selective expression is effected by replacing at least one existing codon of a parent nucleic acid sequence encoding a protein of interest with a synonymous codon to produce a synthetic nucleic acid sequence having altered translational kinetics compared to the parent nucleic acid sequence. The synonymous codon is selected such that it corresponds to an iso-tRNA which, when compared to an iso-tRNA corresponding to the at least one existing codon, is in higher abundance in the target cell or tissue relative to one or more other cells or tissues of the mammal.