NeurosciencebioRxivPreprint
Successful Correction of ALD Patient-derived iPSCs Using CRISPR/Cas9
Eul Sik Jung, Zhejiu Quan, Mi‐Yoon Chang…·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.
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