Functional Cas9 Expressed Internally in AAV Particle

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.

Patents

Patent # Title Country
71961042018280116 Enhanced Modified Viral Capsid ProteinsEnhanced Modified Viral Capsid Proteins JapanAustralia
3,065,946 Enhanced Modified Viral Capsid Proteins Canada
18733483.4 Modified Viral Capsid Proteins Europe
16/619,450 Enhanced Modified Viral Capsid Proteins United States of America

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