Progress in the development of nucleic acid therapeutics.
Abnormal gene expression is a hallmark of many diseases. Gene-specific downregulation of aberrant genes could be useful therapeutically and potentially less toxic than conventional therapies due its specificity. Over the years, many strategies have been proposed for silencing gene expression in a gene-specific manner. Three major approaches are antisense oligonucleotides (AS-ONs), ribozymes/DNAzymes, and RNA interference (RNAi). In this brief review, we will discuss the successes and shortcomings of these three gene-silencing methods, and the approaches being taken to improve the effectiveness of antisense molecules. We will also provide an overview of some of the clinical applications of antisense therapy.
Kalota
A
A
Division of Hematology/Oncology, Department of Medicine, University of Pennsylvania, 421 Curie Blvd, Philadelphia PA, 19104, USA.
Dondeti
V R
VR
Gewirtz
A M
AM
D000818:Animals; D002318:Cardiovascular Diseases / Q000188:drug therapy / Q000473:pathology; D020868:Gene Silencing; D006801:Humans; D007167:Immunotherapy; D007249:Inflammation / Q000188:drug therapy / Q000473:pathology; D009369:Neoplasms / Q000188:drug therapy / Q000473:pathology; D009696:Nucleic Acids / Q000627:therapeutic use*; D016372:RNA, Antisense / Q000627:therapeutic use; D012333:RNA, Messenger / Q000627:therapeutic use*
D009696:Nucleic Acids; D016372:RNA, Antisense; D012333:RNA, Messenger