CRISPR gene editing successfully stopped the progression of Duchenne muscular dystrophy in dogs. It restored muscle function in their hearts, diaphragms and other muscles.
Duchenne muscular dystrophy is caused by a mutation in a gene called dystrophin, which is critical for muscle function. CRISPR was used to restore the function of the mutant dystrophin which returned to 92% of normal in heart muscle.
The researchers who published this study plans to conduct longer-term studies to measure whether the dystrophin levels remain stable and to ensure the gene edits do not have adverse side effects.