Targeting the human genome.

In recent years, some useful nucleic-acid-based tools including antisense oligonucleotides, aptamers, ribozymes, and small interfering RNA have been developed to alter the expression of a given gene. To date, however, these methods have proven to be generally insufficient for many applications and typically have not demonstrated high delivery efficiency or high target specificity in vivo. Emerging technologies that employ artificially designed transcription factors could offer an alternative solution, as they can recognize target DNA sequences with high specificity. In addition, these artificial proteins can be used not only as transcriptional regulators but also as genome modifiers that cleave and stimulate mutations at desired positions in the genome. These nucleotide-targeting molecules must be delivered efficiently to the target cells to promote their therapeutic activity and several delivery technologies have been developed for this purpose.