Efficient manipulation of gene dosage in human iPSCs using CRISPR/Cas9 nickases

Abstract

Ye et al demonstrate a simple and precise method to simultaneously generate iPSC lines with different gene dosages using paired Cas9 nickases. As proof-of-concept they apply this method to examining amyloid precursor protein gene dosage effects in an Alzheimer’s disease patient-derived iPSC line. Their method could potentially advance what we know about disease mechanisms and assist with gene therapy development.