CORDIS Project
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This project aims to enhance gene therapy for blood stem cells by improving gene editing precision using a novel selection strategy. By targeting essential genes, it seeks to enrich cells that have undergone successful gene correction, thereby increasing the effectiveness of treatments for genetic disorders.
Hematopoietic stem and progenitor cell (HSPC) gene therapy involving viral vectors has shown remarkable promise in treating severe genetic disorders.
The advent of gene editing technologies offers new possibilities for broadening the scope of HSPC gene therapy by enabling precise in situ gene correction or targeted transgene integration through homology-directed repair (HDR).
However, HDR efficiency in HSPCs remains limited due to their cell cycle dynamics, suboptimal responses to the repair tem…
OSPEDALE SAN RAFFAELE SRL
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