GeneticsbioRxivPreprint
Correction of a pathogenic mutation in iPSCs derived from a patient with Christianson syndrome using CRISPR/Cas9 genome editing
Li Ma, Qing Wu, Michael Schmidt, Eric M. Morrow
This paper corrects a pathogenic mutation in iPSCs from a Christianson syndrome patient using CRISPR/Cas9, resulting in functional NHE6 protein expression.
DOI 10.1101/2021.05.20.445025