Imagine Institute For Genetic Disease Hopital Necker

The medical center is at the forefront of gene therapy research, particularly focusing on dermatological conditions such as Recessive Dystrophic Epidermolysis Bullosa (RDEB). This rare genetic disorder is characterized by fragile skin that blisters easily, and the center is pioneering innovative treatments to address this condition.

• Ex vivo gene therapy
• Autologous skin equivalent grafts
• Genetic correction using retroviral vectors

Through the use of autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector, the center is advancing the treatment of RDEB. This approach not only aims to improve the quality of life for patients but also sets a precedent for future therapies in genetic skin disorders.