I am Professor at the University of Trento, where I guide a research group focusing on the advancement of genome editing and gene therapy, with a particular interest in gene therapies for the treatment of cystic fibrosis. I earned the degree in Biological Sciences from the University of Genoa in 1990, before moving to the National Institutes of Health (NIH) in Bethesda, MD, to study the molecular biology of retroviruses. In 1998, I was postdoc at Cornell University and subsequently I moved for a Faculty position (Instructor) at the Institute of Gene Therapy at Mount Sinai School of Medicine in New York.

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The Laboratory for Advanced Genome Editing Technologies which I lead at the University of Trento focuses on the development of innovative biotechnology tools, including optimized CRISPR-Cas systems and novel RNA-guided nucleases, for gene therapy applications. To expand the CRISPR-Cas toolbox for we performed a comprehensive screening of a large metagenomic databank from the human microbiome to identify new CRISPR-Cas syste (Pedrazzoli et al, 2023). This search led to the identification of a diverse array of previously uncharacterized CRISPR-Cas effectors and RNA guided nucleases with properties favorable for gene therapy use, such as reduced molecular size and extended target capabilities. We enhanced our computational pipeline to prioritize CRISPR-Cas candidates with known sequence recognition (PAM) and high predicted activity in mammalian cells.

To enable the use of inactive Cas retrieved from the natural reservoir, we developed directed evolution platforms tailored for functional optimization in eukaryotic systems. Our central hypothesis is that genome editing tools originating from bacteria require evolution rounds in eukaryotes to function in mammalian cells. Our work demonstrates that through the evolution platform we obtain Cas variants highly specific and with robust genome editing activity.