Scientists have used silica nanoparticles loaded with DNA to deliver genes safely into mouse brains, a technique that could lead to gene therapies able to repair cells more safely and effectively than current methods, which rely on viral vectors.
Gene therapy seeks to transport genes into the body to treat disorders such as sickle cell anemia, cystic fibrosis, muscular dystrophy and hemophilia by supplementing unhealthy mutant genes with therapeutic proteins. With conventional viral-vector gene therapy, the body’s immune system often reacts and seeks to destroy the transported genes, rendering them ineffective. Moreover, the viral carriers may damage chromosomes when they insert genes.