Figure 1.

The principle of antisense technology, gene therapy and gene editing. In the antisense therapy RNA oligonucleotides (antisense oligonucleotides) are used to inhibit or decrease the protein synthesis by targeting the mRNA of the gene encoding the protein. Gene therapy is based on introducing an additional copy of a healthy gene to restore the cell function. Gene editing technology allows to directly target the DNA sequence of interest and to correct the genomic sequence variant.