A Dual-Vector Ribozyme-Mediated Gene Therapy to Express a Highly Functional Dystrophin Lacking exons 20-50This dual‑AAV vector system is engineered to produce highly functional dystrophin protein equivalents found in asymptomatic or minimally symptomatic individuals with naturally occurring dystrophin exon deletions. |
Tech ID2025-050 College(None) Licensing ManagerInventorsCategories(None) |