Salk researchers have generated disease-free stem cells from patients with mitochondrial disease that can be converted into any cell type including neuronal progenitors (left) or heart cells (right). These could potentially be used for future transplantation into patients.
Credit: Salk Institute
Healthy stem cells differentiate into any cell type for potential cures including brain, muscle, eye and heart cells which would improve the lives of tens of thousands of people around the world with debilitating mitochondrial diseases. “Right now, there are no cures for mitochondrial diseases,” says Prof Juan Carlos Izpisua Belmonte from Salk’s Gene Expression Lab. “Very recently, we’ve developed ways to prevent these diseases, so it was natural to next ask how we could treat them.”
Mitochondrial diseases are caused by any of ~200 mutations that affect the genes of mitochondria. Depending on affected genes and cell types, the diseases can cause muscle weakness, liver disease, diabetes, seizures, developmental delays or vision problems. Existing therapies aim to ease the symptoms or slow the progression of the diseases, but can’t entirely cure them.
Belmonte and collaborators from around the world collected skin samples from patients with mitochondrial encephalomyopathy or Leigh Syndrome, both severe disorders that affect the brain and muscles.
The teams began by using current standard protocols to derive pluripotent stem cells from the skin cells, a process that resets the cells to their most basic state. “During the process of stem cell generation, you spontaneously get different types of clones,”.
METHOD: they move the nucleus of the patient’s skin cells, which contains most of their genes, into a donor egg cell with healthy mitochondria. Then, use the new egg cell to generate pluripotent stem cells. They found the healthy mitochondria took over, and healthy, genetically similar cells from the patient were successfully generated. They can use the healthy cells to generate heart, brain, muscle or eye cells from mutation-free stem cells. But methods to make those cells fully mature and functional and transplant them into patients are still under development. http://www.salk.edu/news/pressrelease_details.php?press_id=2098
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