Aptamers: a new class of oligonucleotides in the drug discovery pipeline?
Aptamers are oligonucleotides identified in a randomly synthesized library containing up to 10(15) 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.
Dausse
Eric
E
Inserm U869, 33076 Bordeaux, France; IECB, University of Bordeaux, France.
Da Rocha Gomes
Sonia
S
Toulmé
Jean-Jacques
JJ
D000818:Animals; D052157:Aptamers, Nucleotide / Q000737:chemistry / Q000494:pharmacology* / Q000627:therapeutic use; D055808:Drug Discovery; D057166:High-Throughput Screening Assays; D006801:Humans; D008268:Macular Degeneration / Q000188:drug therapy / Q000378:metabolism; D009690:Nucleic Acid Conformation; D052156:SELEX Aptamer Technique; D054852:Small Molecule Libraries; D013329:Structure-Activity Relationship; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors
D052157:Aptamers, Nucleotide; D054852:Small Molecule Libraries; D042461:Vascular Endothelial Growth Factor A; C495058:pegaptanib