- 作者: Nanna M Jensen; Trine Dalsgaard; Maria Jakobsen; Roni R Nielsen; Charlotte B Sorensen; Lars Bolund; Thomas G Jensen
- 作者服務機構: Institute of Human Genetics, The Bartholin Building, University of Aarhus, Denmark
- 中文摘要: --
- 英文摘要:
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. - 中文關鍵字: --
- 英文關鍵字: --