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However, h ESC-based treatments involve the controversial use of human embryos and pose a risk of immune-mediated rejection.
Using a patient’s own cells for transplantation would avoid these pitfalls and is possible with induced pluripotent stem cells (i PSCs).
The i PSCs were transduced with CRISPR guide RNAs, Cas9 endonuclease, and a donor homology template.
Despite the gene’s repetitive and GC-rich sequences, 13% of RPGR gene copies showed mutation correction and conversion to the wild-type allele.
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