Strategies for administering targeted therapeutic oligodeoxynucleotides.
Antisense or antigene DNA therapy of diseases that are due to aberrant gene expression is an exciting possibility. A variety of synthetic DNA derivatives has been applied in attempts to regulate the expression of many different genes, both in cell culture and in intact organisms (e.g. mice). Realistic design of human oligodeoxynucleotide-based therapeutic strategies requires many aspects of a candidate disease to be considered (including the disease prevalence, the number and nature of genes and mutations involved, and the tissues which must be targeted). For each DNA derivative intended for therapy, methods of targeting, modes of administration, pharmacokinetics, tissue distribution, toxicity, degradation and excretion must all be considered.