Mr. Bennett, THE USE OF ANTISENSE OLIGONUCLEOTIDES AS THERAPEUTIC AGENTS - A REVIEW, Journal of drug development and clinical practice, 7(3), 1995, pp. 225-235
Antisense oligonucleotides are synthetic nucleotide sequences designed
to inhibit the expression of a single mRNA species. The increasing id
entification of genes associated with disease, and the failure of some
forms of conventional pharmacological agents, has prompted the therap
eutic use of antisense oligonucleotides. Whilst this approach holds gr
eet promise, particularly for diseases in which there is expression of
unique gene sequences, considerable care is required in the design, s
ynthesis and administration of antisense oligonucleotides in this cont
ext. This review summarises the concerns regarding the potential role
of antisense oligonucleotides as therapeutic agents.