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Dog Research on Muscular Dystrophy Can Lead to Cures for Both Dogs and Humans



(p. A13) Researchers used a gene-editing tool to repair a gene mutation in dogs with Duchenne muscular dystrophy, an important step in efforts to someday use the tool to edit DNA in people with the same fatal disease.

In a study published Thursday [Aug. 30, 2018] in the journal Science, researchers at UT Southwestern Medical Center in Dallas and the Royal Veterinary College in London reported that they used the Crispr gene-editing system in four dogs to restore production of dystrophin, a protein crucial for healthy muscle function.


. . .


"It's like putting a good spare tire on a car. It's not as good as the original, but it gets you where you want to go," said Eric Olson, director of UT Southwestern's Hamon Center for Regenerative Science and Medicine and senior author of the paper.

Dr. Olson, who is also founder and chief scientific adviser of Exonics Therapeutics Inc., which licensed the technology from UT Southwestern and helped fund the dog studies, said next steps involve testing Crispr in more dogs and observing them for a year or more. If the approach works in the dogs, he said researchers hope to try Crispr in a clinical trial with people with Duchenne.



For the full commentary, see:

Amy Dockser Marcus. "Gene Editing Shows Promise for Muscular Dystrophy." The Wall Street Journal (Friday, Aug. 31, 2018): A13.

(Note: ellipsis, and bracketed date, added.)

(Note: the online version of the commentary has the date Aug. 30, 2018, and has the title "Crispr Used to Repair Gene Mutation in Dogs With Muscular Dystrophy.")


The study in Science, that is mentioned above, is:

Amoasii, Leonela, John C. W. Hildyard, Hui Li, Efrain Sanchez-Ortiz, Alex Mireault, Daniel Caballero, Rachel Harron, Thaleia-Rengina Stathopoulou, Claire Massey, John M. Shelton, Rhonda Bassel-Duby, Richard J. Piercy, and Eric N. Olson. "Gene Editing Restores Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy." Science (Aug. 30, 2018), DOI: 10.1126/science.aau1549. [Epub ahead of print]






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