On the 20th-22nd of October, 2024 an insightful General Assembly meeting took place in Rome, Italy, kindly hosted by Parent Project aps. The meeting focused on next-generation models and genetic therapies for rare neuromuscular diseases. Some key topics included advanced bioengineered models, novel AAV vector strategies, and updates on muscle-specific lentiviral vectors (LVs). Additionally, there were discussions on gene editing approaches for conditions such as Duchenne Muscular Dystrophy (DMD) and congenital muscular dystrophies (CMD), and the development of preclinical models. Other highlights included insights into human ‘myofiber-on-chip’ models, immune responses to viral vectors, and updates on viral vector technologies. The meeting concluded with a review of ethical projects and future action plans.
