Enhancing ASOs Activity through Genetic Modulation and Small-Molecule Adjuvants in Myotonic Dystrophy Type 1

The seminar, part of the PhD in Translational Medicine, offers an in-depth look at one of the most promising frontiers in biomedical research: improving therapeutic strategies based on antisense oligonucleotides (ASOs) for the treatment of rare neuromuscular diseases.

At the core of the event is a research project focused on Myotonic Dystrophy type 1 (DM1), a complex condition for which the effectiveness of ASOs is often limited by challenges related to their intracellular delivery and activity. The work presented explores innovative approaches to overcome these barriers, combining chemical screening and genetic modulation strategies to identify molecules and cellular mechanisms capable of enhancing ASO uptake and therapeutic efficacy. The goal is to contribute to the development of more effective treatments, with potential applications also for related disorders.

The research is presented by Yasmine Ferchichi, a Marie Skłodowska-Curie PhD candidate at the Universitat de València, whose academic and scientific path spans Italy and international contexts. Her experience, which integrates training in biology with research in neurogenetics and biomedicine, lies at the intersection of basic research and clinical application, offering a dynamic and interdisciplinary perspective.

The seminar thus provides an opportunity for discussion on highly relevant topics in translational medicine, where scientific innovation and therapeutic challenges converge in the search for concrete solutions for diseases that still lack definitive treatments.

Further details are available in the attached poster.