CORDIS Project
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This project aims to develop a gene therapy for Fanconi anemia by correcting hematopoietic stem cells using a novel lentiviral vector. It focuses on mobilizing stem cells from patients and conducting a clinical trial to assess the safety and efficacy of this innovative treatment approach.
Fanconi anemia (FA) is a rare inherited syndrome characterized by the early development of bone marrow failure and increasing predisposition to cancer with age.
Allogeneic hematopoietic cell transplantation (alloHCT) is the only curative therapy for hematopoietic manifestations of FA, although associated with complications arising from myeloablation, graft versus host disease and increased incidence of squamous cell carcinoma.
The genetic correction of autologous hematopoietic stem cells (HSC) w…
CENTRO DE INVESTIGACIONES ENERGETICAS MEDIOAMBIENTALES Y TECNOLOGICAS
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France, Paris
Type: Research institute
Activity type: Research Organisations
SME: No
LONDON
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Spain, MADRID
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
Spain, Barcelona
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
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