Introduction: The capability to translate research to the clinic is contingent upon the ability to study disease-causing mutations from patients in a cell type specific manner. This is most commonly accomplished through the introduction of these disease-causing mutations in induced pluripotent stem cells (iPSCs) and their subsequent differentiation. However, in certain cases, editing these genes […]
2. Improving CRISPR/Cas9 Editing Efficiency to Generate a GBA1 KO due to Pseudogene-induced Homology Directed Repair
