Fabio Dellaquila

Background

About Fabio Dellaquila

I am engaged in the drug discovery pipeline in the newly established Pharmacology department at Selvita in Krakow. My main roles are to support team members, generate preclinical rodent models, and plan and execute PK/PD studies of lead candidate molecules. Previously, I carried my postdoctoral research on CRISPR/Cas9-mediated genome editing delivered by AAV vectors to treat lysosomal storage disorders mouse models and test the safety of in vivo integration. Simultaneously, I researched modified versions of CRISPR/Cas9 to increase integration and expression of a fluorescent reporter in vitro. During my Ph.D. training, I focused on designing and testing gene therapies for two rare inherited retinal disorders — gyrate atrophy of the choroid and retina (GACR) and Usher syndrome type 1B (USH1B). In both projects, I developed and/or tested gene therapy approaches that induced transgene expression and demonstrated improvements in retinal function, morphology, and ultrastructure. The gene therapy for USH1B garnered support from the European Union for translation into clinical applications For this purpose, I selected vector doses in a mouse model that will be translated to humans and, through collaborative efforts with research groups and contract research organizations, we assessed safety, biodistribution, and expression in non-human primates.