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Non-viral episomal modification of cells using S/MAR elements.
Orestis Argyros1, Suet-Ping Wong, Richard P Harbottle
1Imperial College London, Gene Therapy Research Group, Sir Alexander Fleming Building, National Heart and Lung Institute, South Kensington, London SW7 2AZ, UK. o.argyros@imperial.ac.uk
Scaffold matrix attachment regions (S/MARs) elements offer a safer, episomal gene therapy vector alternative to integrated retroviral vectors. These S/MARs-based vectors are efficient, cost-effective, and reduce genotoxicity risks in mammalian cells.
Area of Science:
- Gene therapy vector development
- Episomal DNA maintenance
- Mammalian cell modification
Background:
- Gene therapy shows promise but faces toxicity issues, primarily from integrated retroviral vectors causing oncogenesis.
- Safer, non-genotoxic vectors are crucial for advancing gene therapy.
- Scaffold matrix attachment regions (S/MARs) elements offer a unique class of episomal vectors.
Purpose of the Study:
- To review studies on S/MAR element-based episomal modification of mammalian cells and tissues.
- To highlight applications in liver, brain, muscle, eye, cancer, embryonic cells, and neonatal mice.
- To focus on recent research published between 2000 and 2010.
Main Methods:
- Literature review of studies utilizing S/MAR elements for episomal modification.
- Focus on diverse mammalian cell and tissue types.
- Analysis of data primarily from 2000-2010 publications.
Main Results:
- S/MAR elements enable episomal maintenance of vectors in mammalian cells.
- Successful modification demonstrated across various tissues including liver, brain, muscle, and eye.
- Applications shown in cancer cells, embryonic cells, and neonatal mice models.
Conclusions:
- Vectors containing S/MAR elements provide an efficient method for episomal modification.
- This approach offers a safe and cost-effective alternative for gene therapy vectors.
- S/MAR-based vectors reduce genotoxicity concerns associated with traditional methods.


