Aim of the platform

The gene therapy platform will take advantage of the expertise and recent proof-of-principle data gathered by the consortium partners to establish a unique network of competence for the treatment of neurodegenerative disorder and inherited blindness.
Our main objectives are the following:

  • to validate the approach in models as close as possible to the human condition,
  • to develop vectors and delivery methods applicable to humans,
  • to identify genotyped and phenotyped cohorts of patients and define clinical endpoints to assess treatment efficacy,
  • to establish clinical follow-up and infrastructures fostering the translation of gene therapy into the clinic