CRISPR Nanoparticles Repair 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 open a new way for safer, accurately controlled delivery of gene-editing tools," claims Irina Conboy, Ph.D., a bioengineering professor at the University of California, Berkeley (UC Berkeley), who co-led the CRISPR-Gold research alongside UC Berkeley’s professor Niren Murthy, Ph.D., and collaborators in the U.S. and Japan. "Ultimately, these techniques could be developed into a new medicine for Duchenne muscular dystrophy and a number of other genetic diseases." The authors report their research in Nature Biomedical Engineering , in ...
Original Article: CRISPR Nanoparticles Repair Muscular Dystrophy Gene
