CORDIS Project
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This project focuses on developing gene therapy as a treatment for patients with specific genetic disorders affecting the immune system, such as Wiskott-Aldrich syndrome and sickle cell anemia. It aims to enhance the effectiveness of hematopoietic stem cell transplantation by using genetically modified cells to restore…
Given that (i) not all patients with a monogenic disease affecting the lymphohaematopoietic system have an HLA-genoidentical sibling donor and (ii) severe immunological complications worsen the outcome in HLA-partially-matched hematopoietic stem cell transplantation (HSCT), the genetic modification of autologous hematopoietic stem cells (HSCs) has become a powerful curative treatment option for these individuals.
The present project seeks to further consolidate the rationale for replacing HLA-pa…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France, Paris
Type: Research institute
Activity type: Research Organisations
SME: No
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