Stem cell-derived transplantation offers the hope of curing a range of diseases that more conventional therapies cannot. By using a patient's own stem cells as a source of tissue, certain diseases associated with damaged cell types and tissues could be treated and ultimately reversed. As opposed to using human embryonic stem cells for treatment, which is politically and ethically controversial, this method uses induced pluripotent stem cells (iPSCs) that are derived from the patient. However, in the case of genetic degenerative disorders, patient-derived iPSCs would still contain the harmful genetic mutation, making iPSCs useless as a source of transplant material.
Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 has been suggested as a potential method for fixing genetic mutations in patient-derived iPSCs and thus making the cells suitable for therapeutic transplantation. Authors Alexander Bassuk (University of Iowa, Iowa City) et al. have shown that CRISPR/Cas9 can be used to repair pathogenic mutations in iPSCs derived from a patient suffering from retinitis pigmentosa (Sci. Rep. 6, 19969; 2016). Fibroblasts from the patient's skin were transduced to produce iPSCs, and the genetic mutation causing retinitis pigmentosa was then repaired using CRISPR/Cas9 applied in vitro. Although only 13% of the faulty gene copies were repaired, this proof-of-concept paper moves iPSC treatment a small step closer to the clinic. DMG
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