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Analysis of plasmid DNA from a pharmaceutical perspective
C R Middaugh1, R K Evans, D L Montgomery
1Department of Pharmaceutical Chemistry, University of Kansas, Lawrence 66047, USA.
Journal of Pharmaceutical Sciences
|March 31, 1998
Summary
Future gene therapies and vaccines using plasmid DNA drugs can move beyond animal testing. Advanced analytical methods, including microchip devices and various spectroscopic techniques, offer precise identity and potency testing for polynucleotide drugs.
Area of Science:
- Biotechnology
- Pharmaceutical Science
- Molecular Biology
Background:
- Plasmid DNA is increasingly used as a drug substance for gene therapy and vaccines.
- Current methods for assessing drug substance identity and potency rely on biological (cell or animal) assays.
Purpose of the Study:
- To propose advanced analytical methods for characterizing plasmid DNA drug substances.
- To discuss the chemical stability of plasmids and compare them to protein-based pharmaceuticals.
Main Methods:
- Review of microchip-based mutation detection devices.
- Exploration of chromatographic, electrophoretic, hydrodynamic, and spectroscopic techniques.
- Consideration of polynucleotide sequence analysis versus protein tertiary structure.
Main Results:
- Biological assays can be supplemented or replaced by a suite of advanced analytical techniques.
- These methods offer rigorous assessment of plasmid DNA identity and potency.
- Polynucleotide sequence is a critical determinant of function, analogous to protein tertiary structure.
Conclusions:
- A shift towards advanced analytical methods for plasmid DNA drug substances is feasible and desirable.
- These methods promise more precise, potentially faster, and ethically sound characterization.
- Understanding polynucleotide sequence is paramount for the development of novel therapeutics.