Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34⁺ Hematopoietic Stem and Progenitor Cells.

ABSTRACT: Genome-editing technologies are currently being translated to the clinic. However, cellular effects of the editing machinery have yet to be fully elucidated. Here, we performed global microarray-based gene expression measurements on human CD34⁺ hematopoietic stem and progenitor cells that underwent editing. We probed effects of the entire editing process as well as each component individually, including electroporation, Cas9 (mRNA or protein) with chemically modified sgRNA, and AAV6 transduction. We identified differentially expressed genes relative to control treatments, which displayed enrichment for particular biological processes. All editing machinery components elicited immune, stress, and apoptotic responses. Cas9 mRNA invoked the greatest amount of transcriptional change

SUBMITTER: Cromer MK 

PROVIDER: S-EPMC6171165 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature