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CRISPR editing therapy for Duchenne Muscular Dystrophy 2

Part 2 of 2
Published on November 30, 2022   27 min

Other Talks in the Series: Gene-Drives and Active Genetics

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0:00
Thank you for joining me in Part 2 of the talk on "AAV-CRISPR Therapy for Duchenne Muscular Dystrophy".
0:10
One of the issues with CRISPR Cas9 therapy is the immune response because Cas9 was a protein derived from bacteria, and the way that this bacterial protein will cause immune response when delivered to humans is unclear. In the previous study, in the dystrophic dog model, it was found that Cas9 did not induce an immune response. So to thoroughly study, this issue, we performed a comprehensive study using several different Duchenne muscular dystrophy canine models.
0:57
As I mentioned that immunity to Cas9 has been the concern starting from early on when the CRISPR was proposed as a potential genetic therapy for human diseases.
1:14
But when we did CRISPR editing therapy in a mouse, we found that edited cells can last for up to 18 months. So if there's an immune response, we would expect the edited muscle would be eliminated by the immune response, and several other groups from Charlie Gersbach's lab and Eric Olsen's lab also showed that a single CRISPR editing therapy can result in long-term additive effect. In the mice, it does not cause an immune response. But what about in dogs? This group
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CRISPR editing therapy for Duchenne Muscular Dystrophy 2

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