# of Displayed Technologies: 2 / 2


A Dual-Vector Ribozyme-Mediated Gene Therapy to Express a Highly Functional Dystrophin Lacking exons 19-44
TS-005705 — 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.
  • College:
  • Inventors: Flanigan, Kevin; Gushchina, Liubov; Patrick, Nehaal
  • Licensing Officer: Eidahl, Jocelyn

A Dual-Vector Ribozyme-Mediated Gene Therapy to Express a Highly Functional Dystrophin Lacking exons 20-50
TS-005704 — This 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.
  • College:
  • Inventors: Flanigan, Kevin; Gushchina, Liubov; Patrick, Nehaal
  • Licensing Officer: Eidahl, Jocelyn

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