Yoshioka, Taniguchi, Homma et al. study the effect of AAV-delivered Cas9 and specific guide RNAs to correct the PMP22 gene duplication that causes Charcot-Marie-Tooth disease type 1A. They measured the effect of Cas9 editing in iPSC cells on PMP22 expression and demonstrated how editing reduces cell death and myelination defects in edited iPSC-derived Schwann cells.
- Yuki Yoshioka
- Juliana Bosso Taniguchi
- Hitoshi Okazawa
