CORDIS Project
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This project investigates how gene editing technologies affect hematopoietic stem and progenitor cells (HSPCs), focusing on the DNA damage response and cellular senescence. By identifying factors that hinder HSPC functionality, it aims to improve gene therapy methods for better clinical outcomes in blood disorders.
The success of Hematopoietic Stem and Progenitor Cell (HSPC) gene therapies critically depends on the capacity to genetically engineer HSPCs without compromising their functional properties.
Emerging evidence, including our own, indicates that HSPC exposure to currently available gene transfer and gene editing technologies –which require prolonged ex-vivo culture, high viral vector doses and nuclease-induced DNA double strand breaks– unexpectedly converge on the DNA damage response (DDR), a sign…
OSPEDALE SAN RAFFAELE SRL
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