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DNA and RNA aptamers: from tools for basic research towards therapeutic applications
Henning Ulrich1, Cleber A Trujillo, Arthur A Nery
1Departamento de Bioquímica, Instituto de Química, Universidade de São Paulo, São Paulo, Brazil. henning@iq.usp.br
Combinatorial Chemistry & High Throughput Screening
|October 5, 2006
Summary
Systematic Evolution of Ligands by Exponential Enrichment (SELEX) generates high-affinity aptamers for diverse targets. These nucleic acid aptamers show promise as therapeutic agents, with some undergoing clinical trials and one already FDA-approved.
Area of Science:
- Biotechnology
- Molecular Biology
- Drug Discovery
Background:
- Systematic Evolution of Ligands by Exponential Enrichment (SELEX) is an in vitro selection method.
- SELEX identifies DNA or RNA molecules with high affinity and specificity for various targets, similar to antibodies.
Purpose of the Study:
- To review the applications of SELEX in isolating aptamers.
- To highlight the therapeutic and diagnostic potential of aptamers.
- To discuss advancements and future prospects of aptamers in medicine.
Main Methods:
- SELEX involves selecting oligonucleotide libraries for target binding.
- Chemical modifications enhance aptamer stability and efficacy.
- Automatization of SELEX increases aptamer generation.
Main Results:
- Aptamers have been successfully selected against small molecules, proteins, and whole organisms.
- Numerous aptamers are being evaluated in preclinical and clinical studies.
- Pegaptanib, an anti-VEGF aptamer, is FDA-approved for ocular vascular disease.
Conclusions:
- Aptamers are emerging as a significant class of therapeutic agents.
- SELEX technology has broad applications in developing targeted therapies and diagnostics.
- Further development of aptamers promises advancements in personalized medicine.
