iBET’s gene therapy research awarded at ESACT Frontiers Retreat 2025
Innovative use of 3D-human iPSC-derived models to assess dual-AAV vector system efficiency
Mariana Ferreira, PhD student in the Cell Line Development and Molecular Virology Lab at iBET, won Best Oral Presentation Award at the 2025 ESACT Frontiers Retreat, held in Ljubljana, Slovenia, from June 11th to 13rd.
As part of the award, Mariana was granted an invitation to present her research at ESACT biennial meeting in June 2026, which will take place in Salzburg, Austria.
In the presentation, titled “Assessing the Robustness of Dual-AAV Delivery in Human IPSCs-derived 3D Models”, Mariana shared work performed in collaboration with Professor Francesco Tedesco Stem Cells and Neuromuscular Regeneration Laboratory, in University College of London. The study explores the transduction efficiency of a dual-AAV system developed at iBET, carrying a split-mini-dystrophin gene, in 3D human skeletal muscle models of Duchenne Muscular Dystrophin. This represents one of the first evaluations of dual-AAV transduction in 3D-human iPSC-derived muscle models, offering novel insights that complement the traditional animal model studies and strengthen the translational potential of gene therapies.
The ESACT Frontiers Program, an initiative within ESACT- European Society for the Advancement of Cell Technology, supports early career scientists working in advanced cell technology and related fields. These meetings are focused on career and personal development, networking, and bridging the gap between industry and academia, with scientific-driven as well as career-driven keynote sessions and workshops.
Congratulations to Mariana and the entire research team for this great achievement!
Know more about ESACT Frontiers Retreat 2025 here: https://esact.org/frontiers/frontiers-retreat-2025/
