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