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Development of biotherapies for growth plate disorders

Final Report Summary - GENECELLTHER (Development of biotherapies for growth plate disorders)

This project aimed at developing an innovative therapeutic strategy for achondroplasia, the most common form of short limb dwarfism. Children affected by this rare disease suffer from abnormal long-bone development, resulting in short stature. In the most severe cases, they can suffer from deformations of the skull and vertebrae that can lead to severe neurological and orthopedic complications. Obtained results were highly significant and the recruited researcher successfully developed and tested a recombinant protein approach restoring bone growth in transgenic mice with achondroplasia. These results describe a novel approach for restoring bone growth and suggest that it could be a potential therapy for children with achondroplasia and related disorders.
An engineer and a PhD student were directly recruited from this European funding. The project also triggered the recruitment of 4 Master students and one technician.
The IRG fellowship has contributed to the reintegration of the recruited researcher who was successfully recruited as a senior researcher (position) by Inserm in 2009. This is a permanent position in France, fulfilling the long term integration requirement of the IRG.