Self-amplifying RNA enables rapid, durable, integration-free programming of hiPSCs.

ABSTRACT: Genetic modification of human induced pluripotent stem cells (hiPSCs) is a powerful approach to measure and manipulate the cellular processes underlying differentiation and disease. Conventional genetic engineering of hiPSC lines requires a laborious process involving transfection, selection and expansion that can result in karyotypic abnormalities or transgene silencing during differentiation, limiting their applications. Self-amplifying RNA (saRNA) delivery is a potential alternative integration-free method for durable expression of transgenes. Here, we used saRNA to deliver transcription factors and functional reporters in hiPSCs and demonstrate that expression can persist for weeks. Specifically, saRNA delivery enables highly efficient forward programming to Ngn2-induced neurons and en

SUBMITTER: Della Santina CM 

PROVIDER: S-EPMC12633252 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature