Aptamers: an emerging class of therapeutics

Numerous nucleic acid ligands, also termed decoys or aptamers, have been developed during the past 15 years that can inhibit the activity of many pathogenic proteins. Two of them, Macugen and E2F decoy, are in phase III clinical trials. Several properties of aptamers make them an attractive class of...

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Bibliographic Details
Published in:Annual review of medicine 2005-01, Vol.56 (1), p.555-583
Main Authors: Nimjee, Shahid M, Rusconi, Christopher P, Sullenger, Bruce A
Format: Article
Language:English
Subjects:
Animals
Anti-Infective Agents - therapeutic use
Antidotes - therapeutic use
Aptamers, Nucleotide
Bacterial Proteins - antagonists & inhibitors
Bacterial Proteins - genetics
Clinical trials
Clinical Trials, Phase III as Topic
Disease
DNA-Binding Proteins - therapeutic use
Drug Delivery Systems
Drug Design
Drug Evaluation, Preclinical
Drugs, Investigational - therapeutic use
Gene Library
HIV Infections - drug therapy
Humans
Immune system
Oligonucleotides - genetics
Oligonucleotides - therapeutic use
Proteins
Ribonucleic acid
RNA
Transcription Factors - therapeutic use
Viral Proteins - antagonists & inhibitors
Viral Proteins - genetics
Viruses
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Summary:Numerous nucleic acid ligands, also termed decoys or aptamers, have been developed during the past 15 years that can inhibit the activity of many pathogenic proteins. Two of them, Macugen and E2F decoy, are in phase III clinical trials. Several properties of aptamers make them an attractive class of therapeutic compounds. Their affinity and specificity for a given protein make it possible to isolate a ligand to virtually any target, and adjusting their bioavailability expands their clinical utility. The ability to develop aptamers that retain activity in multiple organisms facilitates preclinical development. Antidote control of aptamer activity enables safe, tightly controlled therapeutics. Aptamers may prove useful in the treatment of a wide variety of human maladies, including infectious diseases, cancer, and cardiovascular disease. We review the observations that facilitated the development of this emerging class of therapeutics, summarize progress to date, and speculate on the eventual utility of such agents in the clinic.
ISSN:0066-4219
1545-326X
DOI:10.1146/annurev.med.56.062904.144915