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
