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Printable Handouts
Navigable Slide Index
- Introduction
- Issues with CRISPR Cas9 therapies
- Immunity to Cas9
- Cas9 immune response in mice
- Cas9 immune response in canines
- CRISPR therapy in 3 canine DMD models
- Canine DMD models: local injection
- CTL response to CRISPR therapy eliminates rescued dystrophin
- Local injection, AAV8, CK8.SpCas9, 1-m-old LRMD, 6-wks post-inj.
- CRISPR rescue correlates with inflammatory cytokine expression
- AAV8 Cas9 expression induces cytotoxic killing of muscle cells in adult normal dogs
- AAV.Cas9 induces a robust cellular immune response in normal puppies
- Cas9-induced cellular response is independent of several factors
- Systemic AAV CRISPR therapy (3m post-inj)
- Systemic AAV CRISPR therapy results in body wide dystrophin rescue
- Systemic AAV CRISPR therapy induces the CTL response in the canine DMD model
- Cas9-specific humoral and cellular immune responses
- Systemic AAV μDys therapy
- Summary (3)
- Acknowledgments (1)
- Acknowledgments (2)
Topics Covered
- Absence of Cas9 immune response in mice
- DMD canine model
- Cas9 immune response in canine
- Local AAV CRISPR therapy in canine DMD models
- Systemic AAV CRISPR therapy in canine DMD models
- Immune response to CRISPR therapy in canine DMD models
Links
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Therapeutic Areas:
Talk Citation
Duan, D. (2022, November 30). CRISPR editing therapy for Duchenne Muscular Dystrophy 2 [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved March 31, 2023, from https://hstalks.com/bs/5134/.Export Citation (RIS)
Publication History
Financial Disclosures
- Prof. Dongsheng Duan has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
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
Transcript
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 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.
0:57
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.
1:14
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