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.
A team of researchers at the University of Wisconsin–Madison is joining the National Institutes for Health (NIH)’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.
The team is led by Shaoqin Gong, a professor of biomedical engineering at the Wisconsin Institute for Discovery (WID) and an expert in creating nanomedicines for human health. She and her collaborators, professor of neuroscience Subhojit Roy, assistant professor of biomedical engineering Krishanu Saha, 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 like Alzheimer’s disease.
“To date, successful genome editing has been mostly mediated by viral vectors,” says Gong, 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 grant from NIH, announced on Tuesday, 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.
Grant: 1UG3NS111688-01 awarded to University of Wisconsin–Madison; ENABLING NANOPLATFORMS FOR TARGETED IN VIVO DELIVERY OF CRISPR/CAS9 RIBONUCLEOPROTEINS IN THE BRAIN