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New approach to treat degenerative eye diseases

By Tim Sandle     Aug 9, 2013 in Science
Scientists have developed a new approach which may one day be able to treat the degenerative eye disease Leber hereditary optic neuropathy (LHON). The new approach borrows from the microbial world.
Mitochondrial gene mutations can lead to a variety of health problems including muscle weakness, heart disease, and blindness. Mitochondria are found within cells and they function to convert fuel from food into a form of energy that cells can use. They also make enzymes for a variety of cell functions.
One such genetic condition is Leber hereditary optic neuropathy (LHON). LHON is a rare disease, occasionally transmitted from a mother to her children. In the affected children it can lead to an acute loss of central vision.
According to a research brief, University of Miami Miller School of Medicine scientists investigated proteins called transcription activator-like (TAL) effectors as part of a search to repair mitochondrial gene defects. In nature, TAL effectors are found only in certain types of plant-infecting bacteria where they enable the bacteria to use plant DNA to multiply and spread infection.
Inspired by this, the researchers began using TAL effectors to modify DNA in a variety of organisms. In one trial, DNA-breaking proteins called nucleases were fused with TALs. The scientists were then able to use these to add or remove specific genes or correct gene mutations (in studies within a test tube).
Given that the most recent studies were similar to what is happening with LHON (the scientists replicated the specific mutation), the researchers hope to one day find a way of reversing the process. A future step will be to undertake some animal studies, to see if the experiments in cells can be replicated in tissues.
The research was funded by the National Eye Institute (NEI), a part of the National Institutes of Health (NIH) and it has been published in the journal Nature Medicine. The study is titled "Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs."
More about degenerative eye, Eye disease, Bacteria, Genetics, Mutation
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