This innovation is a new Econ-skipping gene therapy strategy for Duchenne muscular dystrophy (DMD) that targets exon 33 of the DMD gene. Previous viral DMD therapies rely on micro-dystrophins, which use engineered Isoforms that aren’t naturally expressed by humans, leading to uncertain long term safety and efficacy. To remedy this, development of an AAV-U7snRNA exon-2 skipping vector that restores full-length dystrophin has been demonstrated to be effective. Building on that success, this new innovation uses AAV vectors encoding antisense sequences designed to target exon 33. This is beneficial since it can be used in patients with any frame‑shift mutation affecting exon 33, enabling restoration of the open reading frame and production of a functional dystrophin protein lacking only a single exon.