Researchers at Oregon Health & Science University have made a breakthrough in gene and stem cell therapy that could pave the way for treatments for incurable conditions such as heart disease, diabetes, dementia and other disorders.

They took cells from patients with a genetic disorder and turned them into healthy stem cells, the body's building blocks. They were also able to correct genetic mutations by remaking the cells so that they were able to produce energy, something they could not do before.

They had good results using cells from a variety of patients.

"It's a pretty big advance in generating genetically corrected stem cells," said Shoukhrat Mitalipov, the lead researcher.

In the research, published Wednesday in the journal Nature, Mitalipov and his team studied skin cells from patients with mitochondrial disease, which affects the parts of cells that generate energy. When the mitochondria fail, cells become powerless and whole systems can become dysfunctional. The list of possible symptoms is long, including muscle weakness, visual problems, learning disabilities, heart disease, liver disease, kidney disease, gastrointestinal disorders and dementia.

"Every cell needs energy," Mitalipov said. "The mitochondria should provide it and when they do not, the cells cannot perform their function and disease symptoms start."

About one in 5,000 children are born with mitochondrial disease. Other people develop dysfunction in their mitochondria later in life, leading to chronic conditions.

In the research, the scientists used cells from young patients born with mixture of mutated and healthy mitochondrial genes, putting them in a petri dish to multiply. In the process, the genetic defect disappeared in a few rare instances with the creation of healthy cells. The scientists selected those healthy cells and turned them into stem cells, which could be used to create healthy tissue and eventually whole organs.

The scientists also took cells from patients that only had mutated mitochondria and essentially sucked it out of the cell and replaced it with healthy material from a donor cell.

The next step is treatment, Mitalipov said.

"Now we're working towards treating these patients with their own stem cells," he said. "It's still a long way to go but we're going step by step."

-- Lynne Terry