Title |
An update on targeted gene repair in mammalian cells: methods and mechanisms
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Published in |
Journal of Biomedical Science, February 2011
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DOI | 10.1186/1423-0127-18-10 |
Pubmed ID | |
Authors |
Nanna M Jensen, Trine Dalsgaard, Maria Jakobsen, Roni R Nielsen, Charlotte B Sørensen, Lars Bolund, Thomas G Jensen |
Abstract |
Transfer of full-length genes including regulatory elements has been the preferred gene therapy strategy for clinical applications. However, with significant drawbacks emerging, targeted gene alteration (TGA) has recently become a promising alternative to this method. By means of TGA, endogenous DNA repair pathways of the cell are activated leading to specific genetic correction of single-base mutations in the genome. This strategy can be implemented using single-stranded oligodeoxyribonucleotides (ssODNs), small DNA fragments (SDFs), triplex-forming oligonucleotides (TFOs), adeno-associated virus vectors (AAVs) and zinc-finger nucleases (ZFNs). Despite difficulties in the use of TGA, including lack of knowledge on the repair mechanisms stimulated by the individual methods, the field holds great promise for the future. The objective of this review is to summarize and evaluate the different methods that exist within this particular area of human gene therapy research. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Unknown | 1 | 100% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 1 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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France | 3 | 2% |
Netherlands | 1 | <1% |
Germany | 1 | <1% |
Ireland | 1 | <1% |
South Africa | 1 | <1% |
India | 1 | <1% |
United Kingdom | 1 | <1% |
Spain | 1 | <1% |
Unknown | 126 | 93% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 33 | 24% |
Researcher | 32 | 24% |
Student > Master | 16 | 12% |
Other | 9 | 7% |
Student > Doctoral Student | 7 | 5% |
Other | 23 | 17% |
Unknown | 16 | 12% |
Readers by discipline | Count | As % |
---|---|---|
Agricultural and Biological Sciences | 70 | 51% |
Biochemistry, Genetics and Molecular Biology | 29 | 21% |
Medicine and Dentistry | 11 | 8% |
Physics and Astronomy | 2 | 1% |
Immunology and Microbiology | 2 | 1% |
Other | 6 | 4% |
Unknown | 16 | 12% |