Therapeutic potential of antisense nucleic acid molecules.
Elucidation of many disease-related signal transduction and gene expression pathways has provided unparalleled opportunities for the development of targeted therapeutics. The types of molecules in development are increasingly varied and include small-molecule enzyme inhibitors, humanized antibodies to cell surface receptors, and antisense nucleic acids for silencing the expression of specific genes. This Perspective reviews the basis for various antisense strategies for modulating gene expression, including RNA interference, and discusses the prospects for their clinical use.
Department of Hematology, Pommeranian Medical Academy, ul. Unii Lubelskiej 1, 71245 Szczecin, Poland. jopal@poczta.onet.pl
Joanna B
JB
Opalinska
Alan M
AM
Gewirtz
D000818:Animals; D016375:Antisense Elements (Genetics) / Q000494:pharmacology / Q000627:therapeutic use*; D020868:Gene Silencing / Q000187:drug effects; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000494:pharmacology / Q000627:therapeutic use; D009946:Organothiophosphorus Compounds / Q000494:pharmacology / Q000627:therapeutic use; D013873:Thionucleotides
D016375:Antisense Elements (Genetics); C428115:Olg antisense phosphorothioate oligonucleotide; D016376:Oligonucleotides, Antisense; D009946:Organothiophosphorus Compounds; D013873:Thionucleotides