Marco A Quezada-Ramirez

Sep 10, 2023 | biorxiv.org | Shannon Loncar |Matthew Campbell |Robert Gifford |Marco A Quezada-Ramirez

AbstractGene transfer into hematopoietic stem and progenitor cells (HSPCs) involving indiscriminately integrating viral vectors has unpredictable outcomes including potential adverse events such as leukemogenesis, resulting from insertional mutagenesis. Therefore, identifying and characterizing genomic safe harbor (GSH) sites where exogenous genetic information can be integrated safely into adult progenitor and stem cells is critically important for therapeutic gene addition.