Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations

<div>Mode of gene correction by gene-editing tools. (a) The image is drawn on the basis of the previous report [<a href="/journals/sci/2016/2725670/#B46">19</a>]. The selection cassette (CAG-Neo<sup><i>γ</i></sup>) is inserted into the noncoding regions of the normal BAC DNA by recombineering. The lengths of flanking homology arms are indicated. The gene harboring a mutation base is replaced with the normal BAC DNA by homologous recombination. The selection cassette is removed by Cre recombinase. (b) The programmable nuclease modes, such as ZFN, TALEN, or CRISPR/Cas9, induce DNA double-strand break (DSB) at the target locus. The mode of gene correction of programmable nucleases repairs DSB with the donor DNA, which is additionally introduced.</div>

Stem Cells International

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Figure 1: Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations 

Figure 1 | Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations 