Progetto FRRB ID 4977394- Circulating hematopoietic stem cells as innovative target for gene therapy (cHSPC-GT)
Dati del progetto
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Principal Investigator |
Dr.ssa Serena Scala |
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Ente ospitante |
I.R.C.C.S. Ospedale San Raffaele |
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Area Tematica |
Malattie Rare |
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Data di inizio Progetto |
01/06/2025 |
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Data di fine Progetto |
31/05/2028 |
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Importo assegnato |
500.000,00 € |
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Tipo Progetto |
Individuale |
PROJECT AIMS
Clinical practice would benefit from the identification of new sources of HSPCs for gene therapy of patients with rare diseases diagnosed by neonatal screening, as collection of HSPC by conventional techniques is unfeasible for infants from 0 to 3 months: therefore, the aim of the cHSPC-GT project is to use circulating HSPC cells as an innovative target for gene therapy in patients with rare genetic diseases.