CRISPR gene editing successfully stopped the progression of Duchenne muscular dystrophy in dogs. It restored muscle function in their hearts & more.
www.utsouthwestern.edu
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.
Enjoying this research? Get deeper insights like this delivered every other week.
Every other week our Premium Members receive deep dives like this alongside Rhonda's commentary and 8+ other hand-picked papers.
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.