New gene-editing CRISPR technique successfully corrected mutations in the gene that causes Duchenne muscular dystrophy in heart muscle cells.
www.utsouthwestern.edu
The researchers found that correcting less than half of the heart muscle cells was enough to rescue cardiac function to near-normal levels in the heart tissue.
Duchenne muscular dystrophy leads to degeneration of skeletal and heart muscles. This results in patients being wheelchair-bound and on respirators to breathe. Most people with the disease die by age 30. No cure has been developed.
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Clinical trials expected to start this year.