Precise CCM1 gene correction and inactivation in patient ‐derived endothelial cells: Modeling Knudson's two‐hit hypothesis in vitro

ConclusionWe here demonstrate that a non ‐viral CRISPR/Cas9 approach can not only be used for gene knockout but also for precise gene correction in hard‐to‐transfect endothelial cells (ECs). Comparing patient‐derived isogenicCCM1+/+,CCM1+/ −, andCCM1−/− ECs, we show that the inactivation of the second allele results in clonal evolution of ECs lacking CCM1 which likely reflects the initiation phase of CCM genesis.

Source: Molecular Genetics & Genomic Medicine - May 22, 2019 Category: Genetics & Stem Cells Authors: Stefanie Spiegler, Matthias Rath, Christiane D. Much, Barbara S. Sendtner, Ute Felbor Tags: METHOD Source Type: research