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Comment
. 2010 Jun;18(6):1061-3.
doi: 10.1038/mt.2010.92.

Gene correction in human embryonic and induced pluripotent stem cells: promises and challenges ahead

Kazim H Narsinh  1 , Joseph C Wu
Affiliations
Comment

Gene correction in human embryonic and induced pluripotent stem cells: promises and challenges ahead

Kazim H Narsinh et al. Mol Ther. 2010 Jun.
No abstract available

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Figures

<b>Figure 1</b>
Figure 1
Methods for gene correction in human embryonic and induced pluripotent stem cells. (a) A double-strand break (DSB) induced by ionizing radiation or a zinc-finger nuclease signals recruitment of DNA repair machinery. DNA repair then proceeds by a variety of pathways depending on the cell cycle stage and extent of 3¢ end resection. Homology-directed repair results in successful gene targeting, whereas nonhomologous end joining (NHEJ), single-strand annealing (SSA), or microhomology-mediated end joining (MMEJ) will result in nonspecific mutations. (b) A recombinant adeno-associated virus (AAV) expression vector consists of single-stranded DNA (ssDNA) flanked by palindromic inverted terminal repeats. After removal of the viral capsid, DNA repair machinery components coat the ssDNA to form a nucleofilament structure. Resolution of vector-chromosomal DNA intermediates results in introduction of a targeted modification at the homologous chromosomal locus.
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