Application of antisense technology to therapeutics.
Antisense oligonucleotides inhibit gene expression by binding in a sequence-specific manner to an RNA target. Modern nucleotide chemistry has enabled the synthesis of chemically modified oligonucleotides that are highly resistant to nuclease degradation. Among other applications, these agents are currently being evaluated as potential antiviral and anticancer drugs. However, several unsolved problems remain. Poor efficiency of delivery to cells, tissue toxicity and antisense-independent biological effects of oligonucleotides currently limit the widespread application of antisense oligonucleotides to human disease. This article reviews some of the applications of antisense oligonucleotides and discusses problems associated with these applications.
Heidenreich
O
O
Department of Neuropharmacology, Scripps Research Institute, La Jolla, CA 92037, USA.
Kang
S H
SH
Xu
X
X
Nerenberg
M
M
D000818:Animals; D019380:Anti-HIV Agents / Q000494:pharmacology / Q000627:therapeutic use; D004337:Drug Carriers; D005786:Gene Expression Regulation / Q000187:drug effects*; D015497:HIV-1 / Q000187:drug effects*; D006801:Humans; D008322:Mammals; D016376:Oligonucleotides, Antisense / Q000737:chemistry / Q000494:pharmacology* / Q000627:therapeutic use*; D013873:Thionucleotides / Q000737:chemistry / Q000494:pharmacology
D019380:Anti-HIV Agents; D004337:Drug Carriers; D016376:Oligonucleotides, Antisense; D013873:Thionucleotides