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.

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.

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.

TS-000279 — Inventors at Nationwide Children's Hospital have developed a novel system for importing the CRISPR/Cas9 system into the mitochondria for editing of mtDNA. Pathogenic mutations and deletions in the maternally inherited mitochondrial genome (mtDNA) affect as many as 1/500 births and have poor prognosis for treatment. While the CRISPR/Cas9 system for genomic editing has created a new platform for treatment of genetic diseases, researchers have yet to apply the system to mtDNA due to the challenges of transport into the mitochondria. Inventors at Nationwide Children's Hospital have developed a novel system for importing the CRISPR/Cas9 system into the mitochondria for editing of mtDNA. Benefits: Diseases caused by mitochondrial DNA mutation are difficult to treat. This novel system for CRISPR/Cas9-mediated mtDNA editing provides a platform for treating mitochondrial diseases. State of Development: System has been tested and demonstrated in cell culture systems. We have planned testing of several targets in the mitochondrial genome in both cell culture and in animal models including human cell lines. Inventors have published a study demonstrating the proof of concept (linked below). Potential Applications / Potential Markets: Facilitates rapid and less expensive method of eliminating disease bearing/causing mitochondria. Specifically provides first tool for targeted transport of CRISPR into the mitochondrial nucleus. Using CRISPR has advantages over TALEN endonucleases as they can be introduced into the patient using existing AAV gene therapy vectors. Main application is human gene therapy. IP Status: Patented