The CureCSB project aims to develop a gene therapy by using viral vectors as carriers of the genetic material.

Gene therapy is an approach used to substitute a defective gene through delivery of a functional gene to the target cells. Over the years, the field of gene therapies has grown significantly, with over 4000 clinical trials conducted or ongoing as well as several gene therapy products approved in Europe and the USA, bringing a new hope for patients with incurable diseases. However, the success of a gene therapy highly depends on an effective delivery of the correct gene to the target cells, while promoting a continuous expression of the gene without causing toxic effects. Therefore, functional vectors containing the correct gene must be designed and tested in pre-clinical models for efficacy, biodistribution and toxicity.

Therefore, the goal of CureCSB project is to develop a functional viral vector carrying the CSB gene and test it in CSB disease models to validate its effectiveness and safety profile, with the purpose of translating this gene therapy to the clinical and bring a new future for CSB patients.