Mitochondrial genome mutations are associated with various diseases and gene therapy targeted to mitochondria has the potential to effectively treat such diseases.

A team around Yuma Yamada at Hokkaido University has succeeded to develop a nanocapsule (MITO-Porter)  to deliver a genome editing device directly to mitochondria.

This achievement of a specific genome cleavage within mitochondria is expected to be applied to the treatment of genetic diseases. RNP-MITO-Porter is expected to contribute significantly to the clinical application of mitochondrion-targeted gene therapy.