# of Displayed Technologies: 3 / 3

Applied Category Filter (Click To Remove): CRISPR/Cas9 Gene Editing


Functional Cas9 Expressed Internally in AAV Particle
TS-000598 — CRIPSR/Cas9 gene editing technology is a promising tool for treating disease but requires the delivery of the large Cas9 enzyme. Gene therapy experts at Nationwide Children’s Hospital have taken two different approaches to couple the CRISPR gene editing machinery with AAV, including constructing Cas9 enzyme as a stable component of the AAV particle, as well as expressing Cas9 on the surface of the viral particle.
  • College:
  • Inventors: Loiler, Scott
  • Licensing Officer: Corris, Andrew

AAV-Mediated CRISPR/Cas9 Gene Editing for Correction of DMD Exon Duplications in Patients with Muscular Dystrophy
TS-000438 — Gene therapy experts at Nationwide Children’s have developed an AAV-mediated CRISPR/Cas9 gene editing method for the correction of exon duplications in patients with DMD (Duchenne muscular dystrophy). This therapy has the potential to permanently corrects DMD by stopping and potentially reversing the progression of muscle wasting and fibrosis in affected individuals. Currently about 11% of DMD cases are caused by exon duplications and our experts plan to use this invention to correct for this underlying cause within muscle tissues.
  • College:
  • Inventors: Flanigan, Kevin; Stephenson, Anthony
  • Licensing Officer: Eidahl, Jocelyn

Functional Cas9 Expressed Internally in AAV Particle
TS-000322 — CRIPSR/Cas9 gene editing technology is a promising tool for treating disease but requires the delivery of the large Cas9 enzyme. Gene therapy experts at Nationwide Children’s Hospital have taken two different approaches to couple the CRISPR gene editing machinery with AAV, including constructing Cas9 enzyme as a stable component of the AAV particle, as well as expressing Cas9 on the surface of the viral particle.
  • College:
  • Inventors: Loiler, Scott
  • Licensing Officer: Corris, Andrew

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