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
