The design and development of antisense oligonucleotides and ribozymes for
the treatment of diseases arising from genetic abnormalities has become a r
eal possibility over the past few years. Improvements in oligonucleotide ch
emistry have led to the synthesis of nucleic acids that are relatively stab
le in the biological milieu. However, advances in cellular targeting and in
tracellular delivery will probably lead to more widespread clinical applica
tions. This review looks at recent advances in the in vitro and in vivo del
ivery of antisense oligodeoxynucleotides and ribozymes.