Establishment of a GFP::LMNB1 knockin cell line (CSUi002-A-1) from a dystonia patient-specific iPSC by CRISPR/Cas9 editing

Abstract

LMNB1, as one of the major components of nuclear lamina, anchors heterochromatin and associates with transcription regulation. LMNB1 was previously demonstrated to be upregulated and nuclear-to-cytoplasmic mislocalized in DYT1 dystonia specific neurons. Here, we established a knockin cell line with GFP::LMNB1 fusion expression from a DYT1 patient derived iPSC line, by CRISPR/Cas9 editing. The generated iPSCs displayed GFP and LMNB1 co-localization, reminiscent of successful genomic editing. They remained pluripotent and normal karyotype, and possessed the potential to differentiate into three germ layers. This GFP::LMNB1 knockin iPSC will be used for studying the lamina-pathophysiology of DYT1 dystonia, and other nucleus-centered questions.