New method to closely model diseases caused by splicing defects

Tuesday, August 14, 2012 - 22:00 in Biology & Nature

Researchers have developed a new way of making animal models for a broad class of human genetic diseases -- those with pathology caused by errors in the splicing of RNA messages copied from genes. The new modeling approach can provide unique insights into how certain diseases progress and is likely to boost efforts to develop novel treatments. It was tested successfully in mouse analogs of human spinal muscular atrophy.

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