Generation of a NONO homozygous knockout human induced pluripotent stem cell line by CRISPR/Cas9 editing

The non-POU domain containing octamer-binding gene (NONO) encodes a member of a small family of RNA-binding and DNA-binding proteins, whose variants can cause intellectual disability and congenital heart defects. In this study, we generated a homozygous NONO knockout (NONO-KO) induced pluripotent stem cell (iPSC) line (CMUi002-A-1) using the CRISPR/Cas9-based genome editing system. The gene-edited line had a normal karyotype, expressed pluripotency markers, and was able to differentiate into all three germ layers in vivo. This cell line will provide a platform to study the pathogenic mechanisms of noncompaction cardiomyopathy and neurocyte dysfunction related to NONO mutations.