New nanoparticles deliver therapy throughout the brain and edit Alzheimer's gene in mice

Gene therapies have the potential to treat neurological disorders like Alzheimer's and Parkinson's diseases, but they face a common barrier – the blood-brain barrier.

Now, researchers at the University of Wisconsin-Madison have developed a way to move therapies across the brain's protective membrane to deliver brain-wide therapy with a range of biological medications and treatments.

"There is no cure yet for many devastating brain disorders," says Shaoqin "Sarah" Gong, UW-Madison professor of ophthalmology and visual sciences and biomedical engineering and researcher at the Wisconsin Institute for Discovery. "Innovative brain-targeted delivery strategies may change that by enabling noninvasive, safe and efficient delivery of CRISPR genome editors that could, in turn, lead to genome-editing therapies for these diseases."

In a study recently published in the journal Advanced Materials, Gong and her lab members, including postdoctoral researcher and first author of the study Yuyuan Wang, describe a new family of nanoscale capsules made of silica that can carry genome-editing tools into many organs around the body and then harmlessly dissolve.

By modifying the surfaces of the silica nanocapsules with glucose and an amino acid fragment derived from the rabies virus, the researchers found the nanocapsules could efficiently pass through the blood-brain barrier to achieve brain-wide gene editing in mice.

In their study, the researchers demonstrated the capability of the silica nanocapsule's CRISPR cargo to successfully edit genes in the brains of mice, such as one related to Alzheimer's disease called amyloid precursor protein gene.

Image source: Jeff Miller.