An update on targeted gene repair in mammalian cells: methods and mechanisms

Nanna M Jensen; Trine Dalsgaard; Maria Jakobsen; Roni R Nielsen; Charlotte B Sørensen; Lars Bolund; Thomas G Jensen

doi:10.1186/1423-0127-18-10

An update on targeted gene repair in mammalian cells: methods and mechanisms

Nanna M Jensen, Trine Dalsgaard, Maria Jakobsen, Roni R Nielsen, Charlotte B Sørensen, Lars Bolund, Thomas G Jensen

Department of Biomedicine - Department of Human Genetics

Research output: Contribution to journal/Conference contribution in journal/Contribution to newspaper › Review › Research › peer-review

37 Citations (Scopus)

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.

Original language	English
Journal	International Journal of Biomedical Science
Volume	18
Issue	10
Pages (from-to)	10
ISSN	1550-9702
DOIs	https://doi.org/10.1186/1423-0127-18-10
Publication status	Published - 2 Feb 2011

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title = "An update on targeted gene repair in mammalian cells: methods and mechanisms",

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.",

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AU - Jensen, Nanna M

AU - Dalsgaard, Trine

AU - Jakobsen, Maria

AU - Nielsen, Roni R

AU - Sørensen, Charlotte B

AU - Bolund, Lars

AU - Jensen, Thomas G

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N2 - 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.

AB - 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.

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An update on targeted gene repair in mammalian cells: methods and mechanisms

Abstract

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