A Dual-Vector Ribozyme-Mediated Gene Therapy to Express a Highly Functional Dystrophin Lacking exons 19-44

This dual‑AAV vector system is engineered to produce a highly functional dystrophin protein that mirrors the isoform observed in asymptomatic or minimally symptomatic individuals with naturally occurring deletions across exons 19–44. This approach represents a significant advance over current microdystrophin therapies, such as Elevidys, which have shown limited efficacy and safety issues in certain patient groups.

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