DNA finding could mean new gene therapies

March 12, 2012 at 9:30 PM
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LOS ANGELES, March 12 (UPI) -- U.S. scientists say they've identified, for the first time, a way to fix mutations in human DNA, a finding with implications for treating a host of diseases.

Currently, there is no way to successfully repair or compensate for these mutations in the human mitochondrial genome, implicated in neuromuscular diseases, metabolic defects and aging, researchers at UCLA said.

Scientists at the UCLA stem cell center, and the departments of chemistry and biochemistry and pathology and laboratory medicine, said targeting corrective, or messenger RNAs may correct mutations in human mitochondrial DNA.

RNA molecules play an active role in cells by catalyzing biological reactions, controlling gene expression and directing the synthesis of proteins.

"I think this is a finding that could change the field," Dr. Michael Teitell, a professor of pathology and laboratory medicine, said. "We've been looking to do this for a long time and we had a very reasoned approach, but some key steps were missing.

"Now we have developed this method and the next step is to show that what we can do in human cell lines with mutant mitochondria can translate into animal models and, ultimately, into humans."

Mitochondria generate most of the energy supply within a cell and are also are involved in other cellular processes, including signaling, differentiation, death, control of the cell cycle and growth, the researchers said.

The findings could lead to a form of gene therapy by compensating for mutations that cause a wide range of diseases, study co-senior author Koehler.

"This opens up new avenues to understand and develop therapies for mitochondrial diseases," researcher Carla Koehler said. "This has the potential to have a really big impact."

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