| 摘要 |
The present invention relates to a method of generating induced pluripotent stem cells (iPSCs), for gene therapy, from a patient-derived somatic cell line, through a one-step method combining iPSC generation factors and gene therapy factors. The method for generating genetically modified iPSCs, through the one-step, can generate gene-corrected iPSCs or disease model iPSCs having undergone mutagenesis by simultaneously introducing, into host cells, a reprogramming episomal vector, which induces the reprogramming of adult somatic cells, and a carrier for gene correction or mutagenesis. The gene-corrected or mutated iPSCs exhibit features identical with and different from other kinds of normal iPSCs, and thus can be used in screening for disease treatment cellular therapeutic agents or for therapeutic substances. |
