Francesca Tavolaro

DC9: Targeting of nucleic acid drugs: a new mitochondrial RNA therapeutic strategy
About me

I pursued my studies in Biotechnology at the renowned Università Statale di Milano, specialising in Pharmaceutical Biotechnology from 2016 to 2020. My journey into the world of research began during my Bachelor’s internship where I was involved in the study of SBMA (spinal bulbar muscular atrophy), which sparked my interest in neuromuscular diseases.

Eager to deepen my involvement in research, I successfully obtained in 2023 a Master’s Degree in Medical Biotechnology and Molecular Medicine at the esteemed Università Statale di Milano, focusing on Personalized Medicine and Diagnostics.

During my one-year Master’s internship at Ca’ Granda General Hospital of Milan, my fascination with mitochondrial DNA grew, leading me to work on mitochondrial disorders. There I had the chance to contribute to the discovery of a novel mutation in a gene involved in the assembly of the mitochondrial respiratory chain, leading to isolated complex IV deficiency in a pediatric patient with hypertrophic cardiomyopathy and hypotonia. This experience helped me a lot shaping my research skills and fueled my passion for mitochondrial disorders.

I am now enthusiastic about embarking on a PhD journey as part of the MITGEST project, aiming to expand my knowledge in a field that I’m passionate about and make significant contributions to the advancement of innovative treatments for mitochondrial diseases.

About my project

In this project, using baseclick’s patented click chemistry technology in association with different conjugation technology, we will explore the targeting of RNA/oligonucleotides to the mitochondrial matrix. Furthermore, we will also pursue the conjugation of mitochondria-targeting peptide sequences from nuclear genes encoding mitochondrial proteins to deliver oligonucleotides to the mitochondrial matrix. These mitochondria-targeting agents will be combined with known cellular targeting agents, such as lipid nanoparticles or receptor-specific ligands, and will be tested in cellular contexts for the ability to target oligonucleotides to the mitochondrial matrix.