Mitochondrial disease has been one of the toughest conditions in medicine. It primarily impacts children inheriting the condition, leaving few if any treatment options. Today, a collaborative group of researchers -- including those from Mayo Clinic's Center for Regenerative Medicine, report they have found a way to eliminate the fatal mutations the inherited DNA leaves in cells. The findings appear in the journal Nature.
This is a proof-of-concept study, but it paves the way toward the long search for a therapy.
The announcement from the Oregon University of Health & Science describes the breakthrough:
"The scientists successfully used mitochondrial replacement to create an embryonic stem cell with healthy mitochondria from a patient's skin cell containing mitochondrial DNA mutations. These mutations can cause a vast range of fatal or severely debilitating diseases, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia, and several other neurological diseases. In the United States, 1,000 to 4,000 children are born with mitochondrial DNA disease each year."
Read more about the discovery at Mayo Clinic News Network.