Successful Correction of ALD Patient-derived iPSCs Using CRISPR/Cas9
Eul Sik Jung, Zhejiu Quan, Mi‐Yoon Chang, Wonjun Hong, Ji Hun Kim, Seung Hyun Kim, Seungkwon You, Dae‐Sung Kim, Jiho Jang, Sang‐Hun Lee, Seokjoong Kim, Hoon‐Chul Kang
This study corrects an ABCD1 mutation in ALD patient-derived iPSCs using CRISPR/Cas9 and homology-directed repair, restoring normal protein expression but not fully correcting metabolic issues.
bioRxiv · Feb 23, 2020 · PreprintNeuroscience