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Stem Loop RNA Mediated Transport of Mitochondria Genome Editing Molecules (Endonucleases) into the Mitochondria

詳細技術說明
Inventors at Nationwide Children's Hospital have developed a novel system for importing the CRISPR/Cas9 system into the mitochondria for editing of mtDNA.
*Abstract

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.

*Inquiry
Kyle Murrah, Ph.D. The Research Institute at Nationwide Children's Hospital Office of Technology Commercialization 700 Children's Drive Columbus OH 43205 T: 614 355-4551 F: 614 722-2716 Kyle.Murrah@nationwidechildrens.org
*IP Issue Date
None
*IP Type
Provisional
國家
United States
申請號碼
62/423,140
國家/地區
美國

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