0:00
Hi. My name is Dongsheng
Duan from 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 is my disclosures.
0:33
Duchenne muscular dystrophy
was got his name Duchenne,
who is a French physician.
In 1868, he published
a paper describing
the key boys that have
difficulties in their
walking ability
and the movement ability.
Those kids lost walking ability
and become wheelchair bound
about they're teenagers
and because of Dr. Duchene's
seminal contribution.
So this a disease got the name
of Duchenne muscular dystrophy.
This side, 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 Locuncas group,
that turns out to be one of
the largest gene in the genome.
It has a size of 2.4 Mb with
some 79 exons and many isoforms.
The dystrophin protein is
about 427 kilodaltons.
