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, 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.
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CRISPR editing therapy for Duchenne Muscular Dystrophy 1

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