Antisense molecules for targeted cancer therapy. Scientific aspects and clinical perspectives

摘要

abstract_textpThe efficacy of traditional anti-cancer agents is hampered by toxicity to normal tissues. Recent advances in molecular oncology research have led to the identification of signaling pathways and their regulators implicated in tumor development and progression. Consequently, novel biological agents were designed which specifically target key regulators of the malignant process and are superior to unspecific cytotoxic agents. Antisense molecules comprising conventional single-stranded oligonucleotides (ASO) and small interfering RNA (siRNA) inhibit gene expression mainly on the transcript level. Thus, they specifically target the genetic basis of cancer and are particularly useful for inhibiting the expression of oncogenes the protein products of which are inaccessible to small molecules or inhibitory antibodies. Although antisense oncology trials could not fulfill all expectations so far, recent progress in gene expression profiling and nucleic acid development have raised new hopes that this fascinating gene targeting concept will eventually translate into enhanced clinical efficacy./p/abstract_text