Nanotechnology for genome editing in multiple muscles simultaneously

Many intractable diseases are the result of a genetic mutation. Genome editing technology promises to correct the mutation and thus new treatments for patients. However, getting the technology to the cells that need the correction remains a major challenge. A new study led by CiRA Junior Associate Professor Akitsu Hotta and in collaboration with Takeda Pharmaceutical Company Limited as part of the T-CiRA Joint Research Program reports how lipid nanoparticles provide an effective means for the delivery to treat Duchenne muscular dystrophy (DMD) in mice.