San Raffaele Telethon Institute for Gene Therapy

Epigenetic regulation and targeted genome editing


Group Leader

Angelo Lombardo


Our Group has pioneered the field of targeted genome editing with artificial nucleases, steadily contributing to its advances for the treatment of human diseases. During these studies, we have also provided experimental frameworks and tools that are now widely adopted by the gene editing community. By capitalizing on these achievements, we have also developed a novel gene silencing modality that exploits epigenetics, rather than genetic inactivation or post-transcriptional down-regulation, to permanently repress genes of therapeutic.

Research activity

Our research efforts revolve around two main areas.

  1. Targeted Epigenetic Silencing. This wide-encompassing Area is rooted on the epigenetic silencing technology and aims at realizing its full clinical potential, for both inherited and acquired diseases. Although translationally focused, this area foresees several technological improvements, some of which of potential broad relevance in the field of gene therapy.
  2. Gene discovery. This steadily expanding Area aims at identifying novel players involved in fundamental biological processes, such as epigenetics and gene function in vivo. In the attainment of this goal, it also foresees to disseminate new experimental paradigms and technological advances in the field of genetic screens, the leading technology that will be used here to interrogate, at the genome-scale level and in an unbiased manner, gene function in vitro and in vivo.