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- W2017011155 abstract "Targeted gene alteration strategies (TGAs) make use of single-stranded DNA oligonucleotides to correct disease-causing point mutations. The introduced oligonucleotides bind to their genomic target sequence and induce the cell's intrinsic DNA damage-repair system during replication, leading to permanent correction of the mutation.1 TGA technology has become increasingly relevant to translational medicine since the development of cell reprogramming methods to generate induced pluripotent stem (iPS) cells from fully differentiated somatic cells. Hundreds of iPS cell lines have been generated from patients suffering from a wide spectrum of diseases. In the December issue of Science Translational Medicine, Corti et al. reported the successful combination of iPS and TGA technology with therapeutic cell transplantation in a mouse model of spinal muscular atrophy (SMA).2" @default.
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- W2017011155 date "2013-03-01" @default.
- W2017011155 modified "2023-09-26" @default.
- W2017011155 title "Transplantation of Gene-Corrected Motor Neurons as a Therapeutic Strategy for Spinal Muscular Atrophy" @default.
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- W2017011155 doi "https://doi.org/10.1038/mt.2013.23" @default.
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