Researchers at the University of Illinois Urbana-Champaign have developed a new gene editing tool called SPLICER that helps cellular machinery skip parts of genes responsible for diseases like Alzheimer’s. The tool, which uses exon skipping, has shown improved efficiency in reducing the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer’s disease.
SPLICER builds on the popular CRISPR-Cas9 gene editing platform by using newer Cas9 enzymes that do not require a specific DNA sequence to latch on. This allows for greater flexibility and precision in targeting genes, including those associated with Alzheimer’s.
The researchers applied SPLICER to mouse models of Alzheimer’s disease and found that it reduced the formation of amyloid-beta efficiently. The targeted exon was decreased by 25% in the SPLICER-treated mice, with no evidence of off-target effects.
While SPLICER shows promise in treating Alzheimer’s, its limitations are still being explored. The approach can only treat diseases caused by genetic mutations, and it is not a cure-all solution. Further research is needed to ensure the safety of removing targeted exons and to study the disease progression over time.
The National Institutes of Health, the Muscular Dystrophy Association, the American Heart Association, the Parkinson’s Disease Foundation, and the Simons Foundation supported this work. The researchers hope that SPLICER will one day be used to treat a range of genetic diseases, including Alzheimer’s, Parkinson’s, Huntington’s disease, and Duchenne’s muscular dystrophy.
Source: https://news.illinois.edu/view/6367/743872899