10:40 AM
Oct 12, 2017
 |  BC Extra  |  Preclinical News

Team describes MECP2 gene therapy approach for Rett syndrome

In a paper published in Nature, researchers at the University of Edinburgh and colleagues showed that adeno-associated virus (AAV) delivery of a shortened version of the methyl CpG binding protein 2 (MECP2; RTT) gene containing only two key domains of the protein restored Rett syndrome-like neurological defects in mice. The strategy could be used as gene therapy to treat Rett syndrome, a risk factor...

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