CORDIS Project
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This project investigates gene and stem cell therapies to address skeletal dysplasias, focusing on conditions like achondroplasia and Noonan syndrome. By manipulating growth plate chondrocytes, the aim is to restore normal bone development and improve treatment outcomes for affected children.
Skeletal dysplasias are a diverse and heterogeneous group of hereditary disorders characterized by malformations of bone and cartilage.
These conditions affect development patterns in bones of growing children, and the clinical severity ranges from mild short stature to highly disfiguring, and even to lethal forms.
Their treatment is very challenging, typically with no or limited success.
This proposal is designed to explore the use of gene- and stem cell-based approaches to influence bone devel…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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