Nanoparticles deliver gene-editing tools into Duchenne mice
Researchers have developed virus-like nanoparticles capable of delivering gene-editing tools into muscle cells to treat Duchenne muscular dystrophy. This method shows promise in bypassing the limitations of current viral vectors, with potential future applications in neurological disease treatment.
Why it matters
This breakthrough could overcome significant hurdles in gene therapy, such as immune reactions and cargo size limitations, potentially expanding the reach of genetic medicine.
Nanoparticles resembling viruses can carry gene-editing tools into muscle and help restore a critical protein in a mouse model of Duchenne muscular dystrophy, according to a new preprint .
The approach currently works for muscle. But the team behind the work is exploring similar delivery options that could help treat a range of conditions, including neurological diseases.
“We have to understand how well it will get into the brain and also how well it can get into specific types of neurons,” says study investigator Feng Zhang , professor of neuroscience, brain and cognitive sciences and biological engineering at the Massachusetts Institute of Technology.
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