The laboratory of Gene Editing for Rare Diseases, led by Prof. Dr. Alessia Cavazza, pioneers next-generation genome editing technologies to confront the molecular causes of rare genetic disorders and malignancies. Leveraging long-standing expertise in gene therapy and stem cell biology, the team applies state-of-the-art editing tools, including Base and Epigenome Editing, across ex vivo and in vivo models of blood and metabolic disorders.
Director: Prof. Dr. Alessia Cavazza