A5057545075- CAR-T cell therapy research
- Virus-based gene therapy research
- Pluripotent Stem Cells Research
- Hematopoietic Stem Cell Transplantation
The San Raffaele Telethon Institute for Gene Therapy
2022-2023
Istituti di Ricovero e Cura a Carattere Scientifico
2022-2023
Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele
2022-2023
Vita-Salute San Raffaele University
2022-2023
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning deplete bone marrow (BM) for modified cells. We show that mobilizers create an opportunity seamless engraftment exogenous cells, which effectively outcompete those repopulate depleted BM. The competitive advantage results from rescue during...