Aptamers: a new class of oligonucleotides in the drug discovery pipeline?

• Published in: Current opinion in pharmacology Vol. 9; no. 5; pp. 602 - 607
• Main Authors: Dausse, Eric, Da Rocha Gomes, Sonia, Toulmé, Jean-Jacques
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.10.2009
• Subjects: Animals; Aptamers, Nucleotide - chemistry; Aptamers, Nucleotide - pharmacology; Aptamers, Nucleotide - therapeutic use; Drug Discovery; High-Throughput Screening Assays; Humans; Internal Medicine; Macular Degeneration - drug therapy; Macular Degeneration - metabolism; Medical Education; Nucleic Acid Conformation; SELEX Aptamer Technique; Small Molecule Libraries; Structure-Activity Relationship; Vascular Endothelial Growth Factor A - antagonists & inhibitors
• ISSN: 1471-4892; 1471-4973
• DOI: 10.1016/j.coph.2009.07.006

Aptamers are oligonucleotides identified in a randomly synthesized library containing up to 1015 different molecules that fold into defined three-dimensional structures. Following their selection for predetermined properties at the end of an iterative process known as SELEX (Systematic Evolution of Ligands by Exponential enrichment) they can be chemically modified in order to provide them with additional properties. These molecules display both high affinity and specificity for their target. Aptamers constitute promising molecules for therapeutic applications as exemplified by pegaptanib, an aptamer-derived anti-VEGF compound shown to be effective in treating age-related macular degeneration.