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CRISPR editing therapy for Duchenne Muscular Dystrophy 2
Published on November 30, 2022 27 min
Other Talks in the Series: Gene-Drives and Active Genetics
Guidance for responsible testing and implementation of gene-drive systems
- Prof. Stephanie James
- Foundation for the National Institutes of Health, USA
Suppressing mosquito disease vectors via manipulating fertility, sex and male-courtship drive
- Prof. Craig Montell
- University of California, Santa Barbara, USA
Thank you for joining me in part 2 of the talk on AAV CRISPR therapy for Duchenne Muscular Dystrophy.
One of the issue 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 the lever to humans is unclear. In the previous study, in the destruct dog model, it was found that Cas9 did not induce immune response. So to soley study, this issue, we perform a comprehensive study using several different Duchenne muscular dystrophy canine models.
As I mentioned that immunity to cas 9 has been the concern starting from early on when the CRISPR was opposed as a potential genetic therapy for human diseases.
But when we did CRISPR agiment therapy in a mouse, we found that edited cell can last for up to 18 months. So if there's immune response, we would expect the edited muscle would be eliminated by immune response, and several other groups from Charlie cost-push lab and Eric Olsen's lab also show that by a single CRISPR editing therapy can result in long-term additive effect. In the mice, it does not cause immune response. But what about dog? This group