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.
