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

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.
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CRISPR editing therapy for Duchenne Muscular Dystrophy 1

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