CRISPR genome modification holds good promise for modifying disease-causing mutations, but it can cut DNA and endanger human health. Researchers in Ohio have succeeded in modifying a basic editor – a technology built on CRISPR – to safely and accurately edit DMD mutations without DNA cleavage in adult mice. Systemic delivery of the adeno-associated virus that carries the editor led to dystrophin repair and improved mouse function. The findings are published in the journal Nature Communications.
The next step in the research is a larger clinical trial in animals. Hahn and his team have been developing treatment strategies for muscular dystrophy for years, and were part of a group that first demonstrated in 2015 that genome modification could be used to modify Duchenne gene mutations in postnatal animals. The study was funded by the US National Institutes of Health and the Upper Muscular Dystrophy Project. Han has applied for a patent based on the results of the study.