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Printable Handouts
Navigable Slide Index
- Introduction
- Disclosures
- Duchenne muscular dystrophy (DMD)
- The DMD gene
- What is the function of dystrophin?
- Loss of dystrophin causes DMD
- CRISPR editing can restore dystrophin
- AAV CRISPR therapy in mdx mice (1)
- AAV CRISPR therapy in mdx mice (2)
- AAV CRISPR therapy in mdx mice (3)
- DMD is a chronic disease
- Systemic AAV CRISPR therapy in mdx mice
- Evaluation of dystrophin expression by immunostaining
- Evaluation of dystrophin expression by western blot
- Heart histology and function
- AAV vector genome copy number
- Repeat systemic CRISPR therapy with more gRNA vector (1)
- Repeat systemic CRISPR therapy with more gRNA vector (2)
- Dystrophin transcript
- Long-term CRISPR therapy: skeletal muscle (1)
- Long-term CRISPR therapy: skeletal muscle (2)
- Long-term CRISPR therapy: cardiac muscle (1)
- Long-term CRISPR therapy: cardiac muscle (2)
- On and off-target CRIPSR editing
- Summary (1)
- DMD is a disease of failed muscle regeneration
- Can AAV9 transduce muscle stem cells? (1)
- Can AAV9 transduce muscle stem cells? (2)
- AAV9 transduced Pax7+ muscle stem cells in Ai14 mice (1)
- AAV9 transduced Pax7+ cells in Pax7-ZsGreen/Ai14 mice
- Can AAV9 transduced muscle stem cells regenerate?
- Grafted muscle must undergo complete necrosis before it regenerates
- Grafted muscle regenerates from its own satellite cells
- Can AAV9 transduced muscle stem cells regenerate?
- One week after grafting
- Six weeks after grafting
- AAV9 can transduce muscle stem cells & regenerate
- Can AAV9 CRISPR therapy correct mutations in muscle stem cells? (1)
- Can AAV9 CRISPR therapy correct mutations in muscle stem cells? (2)
- Can AAV vector in the pre-graft lead to CRISPR editing in regenerated graft?
- Minimum amount of AAV Cas9 vector needed to achieve CRISPR editing
- Molecular confirmation of edited genome in regenerated grafts
- Sanger sequencing showed similar genome editing in pre-grafts and regenerated grafts
- Confirmation of results
- Summary (2)
Topics Covered
- Duchene Muscular Dystrophy (DMD)
- Dystrophin function
- MDX mouse model
- AAV CRISPR local therapy in mice
- AAV CRISPR systemic therapy in mice
- Myogenic stem cells can be transduced and edited by AAV9 CRISPR therapy
Links
Series:
Categories:
Therapeutic Areas:
Talk Citation
Duan, D. (2022, November 30). CRISPR editing therapy for Duchenne Muscular Dystrophy 1 [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 6, 2024, from https://doi.org/10.69645/PPUK2531.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 1
Published on November 30, 2022
39 min
Other Talks in the Series: Gene-Drives and Active Genetics
Transcript
Please wait while the transcript is being prepared...
0:00
Hi. My name is Dongsheng
Duan, I am from the
Department of Molecular
Microbiology and Immunology
at the University of Missouri
in Columbia, Missouri.
The topic I'm going to
share with you today is
"CRISPR Editing Therapy for
Duchenne Muscular Dystrophy."
This is Part 1 of my talk.
I look forward to see you in
the second part of my talk.
0:29
Here are my disclosures.
0:33
Duchenne muscular dystrophy
got his name from Duchenne,
who is a French physician.
In 1868, he published
a paper describing
the key boys that have
difficulties in their
walking ability
and their movement ability.
Those kids lost their
walking ability
and became wheelchair-bound
when they became teenagers.
And because of Dr. Duchene's
seminal contribution,
so this disease got the name
of Duchenne muscular dystrophy.
On this slide, you can see
a boy who is 12 years old,
who has Duchenne
muscular dystrophy
and he cannot walk anymore.
1:21
The gene which its
mutation cause Duchenne
muscular dystrophy,
was discovered in 1987
by L. Kunkel's Group,
it turns out to be one of
the largest gene in the genome.
It has a size of 2.4 Mb with
79 exons and many isoforms.
The dystrophin protein is
about 427 Kilodaltons.