Sequential factor delivery enables efficient workflow for universal gene editing in clinical grade iPS cells.

ABSTRACT: Human induced pluripotent stem cells (iPSCs) are gaining momentum as a powerful starting material in cell therapy. To fully harness their potential, CRISPR technology permits endogenous gene modifications as well as the introduction of advanced features, to increase the immune compatibility of the cells or insert suicide genes for enhancing therapeutic safety, for instance. However, genetic manipulation of iPSCs, in particular the generation of knock-in lines, remains relatively inefficient. Conventional mitigation strategies, such as enriching for positive cells using antibiotic selection or complex instrumentation, may, however, cause conflicts with good manufacturing practice (GMP) requirements. To address this challenge, we have systematically optimized a basic gene editing procedure u

SUBMITTER: Berger T 

PROVIDER: S-EPMC12432238 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature