Our expanding knowledge in genetics, gene expression regulation,
and availability of many excellent genetic tools has rendered principally
the whole genome and its products amenable for therapeutic
intervention with nucleic-acid-based molecules. In the light of this,
many gene therapeutic approaches have arisen, from classical gene replacement
therapy to more novel antisense-based methods, aiming at
altering gene expression by various means, including by expressing/
correcting deficient genes, interfering with transcription, pre-mRNA
splicing, mRNA catabolism, or translation. Collectively, different classes
of nucleic acids and their synthetic ON analogs hold enormous clinical
potential for the treatment of disease