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The project IMPROVING-GT focuses on enhancing gene therapy for hematopoietic stem/progenitor cells by improving cell engraftment and reducing toxic conditioning requirements. It aims to develop safer and more effective treatments for blood disorders, potentially expanding the therapy's applicability to a wider range of…
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) refers to the transfer of nucleic acids into HSPCs, to either add a new copy of a “healthy” gene or to correct a mutated gene. HSPC-GT is successfully used in clinic to treat patients with hematopoietic malignancies and several inherited diseases of the hematopoietic system.
Yet, its current use is accompanied by acute conditioning-related toxicities, which impose a burden on patients and limit its application to the most severe condition…
OSPEDALE SAN RAFFAELE SRL
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