Cell and Gene Therapy

Rett syndrome: advances in gene therapy brings hope

Published March. 26. 2024
CGT Banner -Rett syndrome

The FDA approved trofinetide about a year ago as the first drug for treating Rett syndrome, a rare genetic disorder caused by mutations in the MECP2 gene. Researchers in Shanghai, China are exploring a novel gene therapy approach for Rett syndrome using direct injection of normal MECP2 genes enclosed in a viral vector. Unlike traditional methods, this approach aims to avoid liver toxicity and reduce costs by skipping cell alteration in a lab. Initial experiments in mice show promising results, with increased protein levels in the brain, although some regions responded better than others. This innovative approach holds potential for developing a more affordable gene therapy option fr Rett syndrome patients


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