He explored the possibility of using gene therapy or gene editing—technologies that were dominating headlines for their ability to tackle other rare genetic disorders. But scientists told him those approaches would be difficult to implement for Dravet. Instead, a newfangled idea called transfer RNA (tRNA) therapy seemed like it might be the answer.
Drug Discovery tRNA therapies could help restore proteins lost in translation.
A new class of therapies based on transfer RNA could treat forms of cystic fibrosis, muscular dystrophy, genetic epilepsies, and more by.
Ryan Cross
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