Cholesterol is a fundamental component of the brain that contributes to all cognitive processes such as learning and memory. In patients with neurodegenerative diseases, the metabolism of brain cholesterol is disrupted. Dr. Nathalie Cartier-Lacave, a physician-researcher and gene therapy pioneer, and her team are conducting research to develop gene therapy treatments for neurodegenerative diseases, particularly Huntington's and Alzheimer's disease. These diseases, for which there is no effective treatment, lead to a deterioration in the functioning of nerve cells. On a daily basis, they manifest themselves through severe cognitive or motor symptoms that lead to death. Their family, social and economic impact is major.
To restore the metabolism of cerebral cholesterol, the team has developed an innovative technology based on gene therapy tools to introduce an enzyme into the brain. Similar to Trojan horses, these AAV vectors can bring a gene into the cell and restore or provide a function. Ultimately, this approach protects neurons and stops the progression of these diseases.
Erganeo supports and accompanies the project based on work initiated at the Faculty of Pharmacy of Paris Descartes University and aimed at developing a new therapeutic approach against Huntington's and Alzheimer's diseases. Erganeo has made available its expertise as well as its human, technical and financial resources to ensure the protection, maturation and valorization of the project's research results.
Nathalie Cartier-Lacave testifies in the program Vivement Dimanche broadcast last January 26.
