AAP Développement de modèles expérimentaux pour les maladies rares – Fondation Maladies Rares
Genome editing and advances in associated methodologies represent a technological revolution that extends opportunities in rare diseases research and new avenues towards addressing pathophysiological and therapeutic approaches, by allowing the development of models for the investigation of human diseases. This call aims to give a significant boost to the development of new experimental models of rare diseases, in order to:
- improve the understanding of molecular and cellular mechanisms leading to pathological conditions related to a rare disease,
- provide evidence for therapeutic proofs of principle that may eventually lead to treatment.