The recent advent of induced pluripotent stem cells (iPSCs) and gene therapy tools has raised the possibility of autologous cell therapy\nfor rare genetic diseases.However, cellular reprogramming is inefficient in certain diseases such as ataxia telangiectasia, Fanconi\nanemia, LIG4 syndrome, and fibrodysplasia ossificans progressiva syndrome, owing to interference of the disease-related genes. To\novercome these therapeutic limitations, it is necessary to fundamentally correct the abnormal gene during or prior to the reprogramming\nprocess. In addition, as genetic etiology of Parkinson�s disease, it has been well known that induced neural stem cells (iNSCs)\nwere progressively depleted by LRRK2 gene mutation, LRRK2 (G2019S).Thus, to maintain the induced NSCs directly derived from\nPD patient cells harboring LRRK2 (G2019S), it would be ideal to simultaneously treat the LRRK2 (G2019S) fibroblast during the process\nof TD. Therefore, simultaneous reprogramming (or TD) and gene therapy would provide the solution for therapeutic limitation\ncaused by vulnerability of reprogramming or TD, in addition to being suitable for general application to the generation of autologous\ncell-therapy products for patients with genetic defects, thereby obviating the need for the arduous processes currently required.
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