Doctors have limited options for treating mitochondrial disease, but a team led by a prominent innovator may have taken an important step toward developing new therapies: rewinding diseased cells from patients to create pluripotent stem cells with healthy mitochondrial DNA.
Cells like these, which potentially could be reprogrammed to develop into a variety of different cell types, could someday be the basis for individualized transplant therapies that could counteract some of the severe impairments mitochondrial disease inflicts, including cardiac, vision and neurological woes.
"This is a first step toward regenerative medicine [for mitochondrial disease patients]," said Oregon Health and Science University stem cell researcher Shoukhrat Mitalipov, lead author of a study describing the new research that was published Wednesday in the journal Nature.