Genome editing based on programmable nucleases offers the possibility to change, in a targeted manner, the genetic information in eukaryotic cells. Despite this, there are many crucial aspects underlying these technologies that require further research and refinement. These aspects include devising improved methods for delivering the attendant molecular tools into target cells; and increasing the efficiency of precise genome editing procedures. My Ph.D. research will focus on improving: (i) the delivery of GE tools by testing high-capacity adenoviral vectors, (ii) the genome editing precision by investigating the utility of nicking, as opposed to breaking, CRISPR-Cas9 “nickases” and (iii) the efficiency of genome editing by investigating the concept of tethering-assisted GE. This research will be carried out using Duchenne muscular dystrophy as a target disease model and human muscle progenitors as target cells.
In October 2016, I obtained my M.Sc. degree in Cellular and Molecular Biotechnology from the University of Trento, Italy. During this Master programme, I had the opportunity to carry out a six-month internship in the Virus and Stem Cell Biology laboratory of the LUMC, under the supervision of Dr. Manuel Gonçalves. The research project consisted of evaluating the efficiency and precision of different CRISPR/Cas9-based genome editing strategies for tagging an endogenous protein involved in the DNA damage response. Currently, I am a Ph.D. student in the aforementioned laboratory of the LUMC supported by a Marie Skłodowska-Curie ETN-training network grant under the H2020 consortium IMGENE - “Improving Genome Editing Efficiency”.