CRISPR Nanoparticles Repair Duchenne Muscular Dystrophy Gene

Scientists have developed a nonviral approach to delivering the CRISPR/Cas9 gene-editing system to cells that when tested in the mdx mouse model of Duchenne muscular dystrophy (DMD) repaired the faulty DMD gene, leading to improved strength and agility and reduced fibrosis. The platform, called CRISPR -Gold, uses gold nanoparticles to encapsulate all the elements needed for CRISPR/Cas9 gene editing and deliver them directly to cells. “CRISPR-Gold and, more broadly, CRISPR-nanoparticles ope