Department of Cellular, Computational and Integrative Biology, University of Trento, Trento, ITALY

Monday 28 October 2019 | 3:00 pm – ICGEB Trieste, ITALY

Unlocking CRISPR technology for precise and efficient genome editing

Host: M. Giacca

Therapeutic applications of Cas9 are still limited primarily by unspecific cleavages and limitations in nuclease delivery. To tackle these issues, we have generated more specific SpCas9 variants through in vivo evolution. The identification of highly specific SpCas9 variants from a random library was obtained through a yeast-based assay allowing simultaneous evaluation of on- and off-target activity. The same yeast platform has been recently used to evolve Cas9 orthologues with decreased molecular weight and enhanced catalytic activity. The power of CRISPR technology is emerging from various successful gene therapy strategies developed for the treatment of genetic diseases. In the lab we have recently demonstrated the efficacy of CRISPR tools in repairing splicing defects in Cystic Fibrosis. Recent data on Cas evolution and CRISPR use in gene therapy will be presented.