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Lab Reports
August 18, 2010

Treating Hereditary Blindness

JAMA. 2010;304(7):733. doi:10.1001/jama.2010.1084

An international group of researchers from Switzerland, France, Germany, Ireland, and the United States has successfully used a gene therapy strategy to treat retinitis pigmentosa in mice (Busskamp V et al. Science. doi:10.1126/science.1190897 [published online June 25, 2010]). The disease, which can lead to incurable blindness, is a form of inherited retinal degeneration resulting from diverse mutations in more than 44 genes expressed in rod photoreceptors.

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