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From selection hits to clinical leads: progress in aptamer discovery
1Department of Biochemistry, Albert Einstein College of Medicine , Bronx, New York, USA.
Molecular Therapy. Methods & Clinical Development
|April 19, 2016
Summary
Aptamers, or nucleic acid-based drugs, are gaining new interest due to chemical design advances. This review discusses aptamer production, focusing on modifications enabling clinical success and future drug development.
Area of Science:
- Biotechnology
- Nucleic Acid Therapeutics
- Drug Development
Background:
- Aptamers, discovered over 25 years ago, have seen limited FDA approval despite their therapeutic potential.
- Recent advances in chemical design and enzymatic production have revitalized interest in aptamer-based therapeutics.
Purpose of the Study:
- To review aptamer production methods.
- To highlight modifications enabling clinical success of early aptamers.
- To discuss advancements crucial for future aptamer drug generations.
Main Methods:
- Review of scientific literature on aptamer production and chemical modifications.
- Analysis of factors contributing to the clinical success of aptamer-based drugs.
- Discussion of emerging technologies in aptamer synthesis and engineering.
Main Results:
- Significant progress in chemical modifications and enzymatic production has enhanced aptamer stability and efficacy.
- Several new aptamer drugs are advancing to clinical evaluation.
- The resurgence in aptamer interest is driven by improved therapeutic profiles.
Conclusions:
- Advances in aptamer production are critical for their successful clinical translation.
- Future aptamer generations are expected to play a significant role in medicine.
- Continued innovation in aptamer design and manufacturing will drive the development of novel therapeutics.
