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Báo cáo y học: "An update on targeted gene repair in mammalian cells: methods and mechanisms"

Tuyển tập các báo cáo nghiên cứu về y học được đăng trên tạp chí y học quốc tế cung cấp cho các bạn kiến thức về ngành y đề tài: An update on targeted gene repair in mammalian cells: methods and mechanisms | Jensen et al. Journal of Biomedical Science 2011 18 10 http www.jbiomedsci.eom content 18 1 10 tì.NSC The cost of publication in Journal of Biomedical Science Is borne by the National Science Council Taiwan JOURNAL OF BIOMEDICAL SCIENCE REVIEW Open Access An update on targeted gene repair in mammalian cells methods and mechanisms Nanna M Jensen Trine Dalsgaard Maria Jakobsen Roni R Nielsen Charlotte B Sorensen 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 ofTGA 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 triplexforming 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. Introduction In the middle of the nineties the field of targeted gene alteration TGA emerged as a possible method to correct diseases caused by single-base mutations 1 2 . Initially the approach focused on stimulating the endogenous gene repair mechanisms using various single- or doublestranded oligonucleotides. These are complementary to part of the targeted gene except for one mismatched base specifically located at the site of the endogenous mutation. Upon cellular introduction these molecules will interact with the targeted gene sequence by different mechanisms. The mismatch .