A Dual-Vector Ribozyme-Mediated Gene Therapy to Express a Highly Functional Dystrophin Lacking exons 19-44This 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. |
Tech ID2025-049 College(None) Licensing ManagerInventorsCategories(None) |