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October 11, 2018

Nanoparticles deliver new possibilities for editing genes in brain cells


The promise of gene editing using CRISPR/Cas9 technology is enormous: scientists believe the technology, which is capable of making precise changes to DNA, could be used to treat many human diseases by fixing deleterious mutations in patients’ genetic codes.

Shaoqin (Sarah) Gong
Shaoqin (Sarah) Gong

Shaoqin “Sarah” Gong, a Vilas Distinguished Professor of biomedical engineering, is leading a team of University of Wisconsin-Madison researchers who are joining the National Institutes for Health’s Somatic Cell Genome Editing Consortium to overcome one of the biggest barriers to such treatments: safely and effectively delivering CRISPR genome editing machinery to the right cells.

Gong, who’s also an investigator at the Wisconsin Institute for Discovery, is an expert in creating nanomedicines for human health. She and her collaborators, assistant professor of biomedical engineering Krishanu Saha, professor of neuroscience Subhojit Roy and Marina Emborg and Jon Levine of the Wisconsin National Primate Research Center, are developing nanoparticles capable of delivering CRISPR to neurons in the brain to treat neurodegenerative diseases such as Alzheimer’s disease.

“To date, successful genome editing has been mostly mediated by viral vectors,” says Gong, describing an approach that carries methodological difficulties and safety risks. Her team is working on efficient, non-viral delivery vehicles that will facilitate safe genome editing in living animals.

The nearly three-year grant from NIH, announced on Oct. 11, 2018, is part of a wider effort to develop quality tools to perform effective and safe genome editing in human patients. The project led by Gong will add technologies to the gene editing toolbox and may give new hope to those suffering devastating neurodegenerative diseases.