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Nucleic acid aptamers for target validation and therapeutic applications
P Shannon Pendergrast1, H Nicholas Marsh, Dilara Grate
1Archemix Corp., 1 Hampshire Street, Cambridge, MA 02139, USA. pendergrast@archemix.com
Journal of Biomolecular Techniques : JBT
|February 8, 2006
Summary
Aptamers, nucleic acid analogs to antibodies, are generated via SELEX. These molecules offer high affinity, specificity, and therapeutic potential for drug development and validation.
Area of Science:
- Biochemistry
- Molecular Biology
- Drug Discovery
Background:
- Aptamers are nucleic acid molecules that function similarly to antibodies.
- They exhibit high affinity and specificity for target molecules, including proteins.
- Aptamers can modulate protein activity upon binding.
Purpose of the Study:
- To highlight the utility of aptamers in target validation for drug development.
- To present aptamers as a novel class of therapeutics.
- To compare aptamer properties with existing therapeutic modalities.
Main Methods:
- Systematic Evolution of Ligands by Exponential enrichment (SELEX) for aptamer generation.
- Characterization of aptamer affinity, specificity, and in vivo half-life.
- Assessment of aptamer toxicity and immunogenicity.
Main Results:
- SELEX yields aptamers with picomolar to nanomolar affinity and high specificity.
- Modified aptamers demonstrate long in vivo half-lives (hours to days).
- Aptamers are non-toxic, non-immunogenic, and produced via scalable synthesis.
Conclusions:
- Aptamers serve as valuable tools for target validation, complementing methods like siRNA.
- They demonstrate that modulating protein activity can yield therapeutic benefits.
- Aptamers bridge the gap between small molecules and biologics as a new therapeutic class.
