Nucleic-acid therapeutics: basic principles and recent applications.
The sequencing of the human genome and the elucidation of many molecular pathways that are important in disease have provided unprecedented opportunities for the development of new therapeutics. The types of molecule in development are increasingly varied, and include antisense oligonucleotides and ribozymes. Antisense technology and catalytic nucleic-acid enzymes are important tools for blocking the expression of abnormal genes. One FDA-approved antisense drug is already in the clinic for the treatment of cytomegalovirus retinitis, and other nucleic-acid therapies are undergoing clinical trials. This article reviews different strategies for modulating gene expression, and discusses the successes and problems that are associated with this type of therapy.
Department of Hematology, Pommeranian Academy of Medicine, Ul Rybacka 1, 71-252 Szczecin, Poland.
Joanna B
JB
Opalinska
Alan M
AM
Gewirtz
D017209:Apoptosis / Q000187:drug effects; D002986:Clinical Trials as Topic; D021881:DNA, Catalytic / Q000627:therapeutic use; D015870:Gene Expression / Q000187:drug effects; D020868:Gene Silencing; D020570:Genes, myb; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*; D012326:RNA Splicing; D016337:RNA, Catalytic / Q000627:therapeutic use; D015398:Signal Transduction / Q000187:drug effects
D021881:DNA, Catalytic; D016376:Oligonucleotides, Antisense; D016337:RNA, Catalytic