The research is on cardiovascular, neurodegenerative, and metabolism-related diseases. Main target genes are vascular growth factors, and LDL receptor in cardiovascular area and cytotoxic genes in cancer therapy.

The group of Akseli Hemminki uses gene therapy and oncolytic viruses to improve the treatment of cancers lacking currently available effective modalities.

Research covers a wide range of diseases along three main tracks: cellular gene therapy, viral vectors and non-viral vectors. The Institute is headed by Prof. Eithan Galun, M.D.

The group headed by Luigi Naldini performs basic research on gene transfer technologies and new gene therapy strategies for immunodeficiencies, lysosomal storage disorders, thalassemia, hemofilia B.

The interest of group of Alberto Auricchio is to use Adeno-Associated Virus (AAV)-mediated gene transfer as potential therapy for ocular and metabolic diseases

The Gene Therapy research group aims to design new anti-cancer therapies using oncolytic adenoviruses and modulation of gene expression, with particular attention to the p53 tumor suppressor pathway.

The department is headed by Prof. Hidde Haisma and is focused on the development of drugs for the therapeutic manipulation of gene activity. The research is focused on cancer and inflammatory diseases.

The laboratory investigates the properties of blood stem cells and is developing cell and gene therapy for blood disorders: Diamond-Blackfan Anemia and Gaucher Disease.

The laboratory is headed by Prof. Dr. Salih Sanlioglu and his team are involved in the development of novel gene therapy methods for patients with type 2 diabetes using lentiviral vectors.