AAV.U7snRNA-Mediated Gene Therapy Vectors to Skip Exon 18 in Patients with DMDThis novel AAV.U7snRNA‑mediated exon‑skipping gene‑therapy platform targets exon 18 of the DMD gene to treat Duchenne and Becker muscular dystrophies. Unlike micro‑dystrophin approaches currently in clinical development, this strategy leverages antisense sequences embedded within optimized U7snRNA cassettes to modulate endogenous DMD splicing and restore native dystrophin expression. |
Tech ID2026-012 College(None) Licensing ManagerInventorsCategories(None) |