Peptide-Mediated Delivery of Antisense Oligonucleotides and Related Material

Despite their simple conceptual basis, antisense strategies turned out more difficult to implement than initially anticipated. Overwhelming enthusiasm in academic laboratories and in the biotechnology industry in the early 1990s has been followed by a wave of skepticism about the real potential of nucleic acidsbased drugs. An antisense oligonucleotide (ON)-based drug has been approved for the treatment of ocular cytomegalovirus infection, and several clinical trials are now well advanced for the treatment of various cancers and infectious diseases (as reviewed in ref. 1). On the other hand, rapid progresses in genome and transcriptome analysis have given a new impetus to the field when it was realized that the antisense approach might be a strategy of choice for functional genomics and for therapeutic target validation.