“I am Filippo from Italy, and I would like to share a little more about myself. I come from a small town in the Venetian countryside called Passarella, where I grew up with my parents and my younger brother. Our house is surrounded by vineyards on one side and a river on the other, so as a country boy, it was impossible not to get close to nature, and luckily, I loved it! Sure, living outside the city has its pros and cons… like tons of insects trying to eat you alive every summer… but somehow, it just felt right for me.

After high school, I discovered a passion for MARVEL comics, and believe it or not, that was the reason I began my adventure in science. You see, I am actually here because of a bet. Yes, a bet! A friend challenged me to find the genes that could give humans superpowers. I know what you are thinking: “If he finds them, I want my superpowers too!!” Well… long story short, I did not find them, but that bet worked, and inspired me to explore the vast world of biology.

Graduating with a Bachelor’s degree in Biotechnology at the University of Udine motivated me to continue my scientific career by pursuing a Master’s degree in Pharmaceutical Sciences at the University of Copenhagen. For my thesis, I collaborated with Professor Claus Storgaard Sørensen at the Biotech Research Institute & Innovation Centre (BRIC), where I explored the usage of a novel combinatorial treatment to inhibit two cell cycle kinases (PKYMT1 and WEE1) for cancer therapy. 

Building on this experience, I am now a PhD candidate in the EFFecT MSCA doctoral network at the Department of Pediatrics and the Department of Human Genetics of the Radboudumc, working in Dr. Alex Garanto’s lab. Here, I focus on exploring the use of antisense oligonucleotides for different inherited metabolic diseases (IMDs).”

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DC1: Development of antisense technology-based strategies for neurometabolic diseases

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Supervisors: Dr. A. Garanto; Prof. D. J. Lefeber

External mentor: Prof. H. Zhou

Host Institute: Radboud University Medical Center, The Netherlands (www.radboudumc.nl)

Secondments planned: Universidad Autónoma de Madrid, Spain; AstraZeneca, Sweden

Doctoral program: PhD program of the Radboud University

Starting date: September 1st, 2025

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Project description:

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Inherited metabolic diseases (IMD) are one of the most significant causes of death in infants and therefore there is an unmet need to further develop therapeutic strategies for these diseases. DC1 will work on the development of an innovative mutation-independent strategy based on antisense technology (single and double stranded antisense molecules) to target the liver and boost gene expression of metabolic genes to restore the biochemical pathway. The designed molecules will be tested in several cellular models (HEK293T, patient and control fibroblasts and iPSC-derived hepatocytes). Once efficacious molecules have been identified, further optimization using chemical modifications will be performed and the metabolic and genetic safety will be tested by untargeted metabolomics and transcriptomics (e.g., possible altered metabolic pathways and off-targets, resulting from the delivery of these molecules, respectively).

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