Efficient gene editing in induced pluripotent stem cells enabled by an inducible adenine base editor with tunable expression.

ABSTRACT: The preferred method for disease modeling using induced pluripotent stem cells (iPSCs) is to generate isogenic cell lines by correcting or introducing pathogenic mutations. Base editing enables the precise installation of point mutations at specific genomic locations without the need for deleterious double-strand breaks used in the CRISPR-Cas9 gene editing methods. We created a bulk population of iPSCs that homogeneously express ABE8e adenine base editor enzyme under a doxycycline-inducible expression system at the AAVS1 safe harbor locus. These cells enabled fast, efficient and inducible gene editing at targeted genomic regions, eliminating the need for single-cell cloning and screening to identify those with homozygous mutations. We could achieve multiplex genomic editing by creating hom

SUBMITTER: Nandy K 

PROVIDER: S-EPMC10713686 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature