Antisense therapy in clinical oncology: preclinical and clinical experiences.
Nucleic acid molecules have emerged as versatile tools with promising utility as therapeutics for human diseases. The specificity of hybridization of an antisense oligonucleotide (AS ODN) to the target mRNA makes the AS strategy attractive to selectively modulate the expression of genes involved in the pathogenesis of malignant or non-malignant diseases. One AS drug has been approved for local therapy of cytomegalovirus retinitis, and a number of AS ODN are currently tested in clinical trials including ODN that target bcl-2, survivin, and DNA methyltransferase. The clinical studies indicate that AS ODN are well tolerated and may have therapeutic activity. In this overview, we summarize therapeutic concepts, clinical studies, and new promising molecular targets to treat human cancer with AS ODN.
Tamm
Ingo
I
Universitätsmedizin Berlin, Charité, Campus Virchow, Department for Haematology and Oncology, Forschungshaus, Room 2.0315, Augustenburger Platz 1, 13353 Berlin, Germany. ingo.tamm@charite.de
Wagner
Mandy
M
D002986:Clinical Trials as Topic; D017726:Cytomegalovirus Retinitis / Q000188:drug therapy / Q000235:genetics; D006801:Humans; D009363:Neoplasm Proteins / Q000037:antagonists & inhibitors* / Q000235:genetics; D009369:Neoplasms / Q000188:drug therapy* / Q000235:genetics; D016376:Oligonucleotides, Antisense / Q000235:genetics / Q000627:therapeutic use*
D009363:Neoplasm Proteins; D016376:Oligonucleotides, Antisense