CORDIS Project
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This project focuses on developing gene therapy strategies for sickle cell disease by using genome editing techniques to correct mutations in hematopoietic stem cells. The aim is to enhance the understanding of how sickle cell disease affects these cells and improve therapeutic approaches for patients.
Sickle cell disease (SCD) is one of the most prevalent monogenic diseases in Europe. A single amino acid substitution in the beta-globin chain of the adult hemoglobin (Hb) drives red blood cell sickling and multi-organ damage.
The clinical severity of SCD is alleviated by the co-inheritance of mutations causing expression of fetal gamma-globin in adult life ? a condition termed hereditary persistence of fetal hemoglobin (HPFH).
Transplantation of autologous, genetically modified hematopoietic st…
IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
Italy, Milano
Type: Company (for-profit)
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: No
Germany, Freiburg
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Israel, Ramat Gan
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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