Due to a genetic defect in one of the cell lines making up the blood, blood disease can occur. Depending on the cell concerned – white blood cells, red blood cells, platelets – this disease takes a wide variety of forms: anemia, mastocytosis, hemophilia, etc. The objectives of this program are to understand the molecular origin and expression of these diseases, and to develop new therapeutic strategies (including gene and cell therapies) to treat them.

It involves:

• 3 research laboratories of the Institut Imagine
• 4 translational research units
• 2 clinical services
• 4 hospital laboratories
• 5 rare disease reference centers
• 1 Labex (excellence laboratory funded by the Investments for the Future program)
• the DIM (Area of Major Interest) for Gene Therapy, funded by the Ile-de-France region, to unite actors in the region and accelerate the development of gene therapy
• 1 Chair

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