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This article on therapeutic applications of RNA editing quotes MBL Senior Scientist Joshua Rosenthal, co-founder of a biotechnology company, Korro Bio, working in this field.

Monica Coenraads admits that as a first-time parent who hadn’t spent much time around children, she was slow to notice that something was wrong with her daughter, Chelsea. By the time Chelsea was 1 year old, however, her development had obviously stalled and even begun to reverse. She only learned to speak one word and soon stopped saying anything. Chelsea could only walk if someone held her upright. She lost the ability to grasp and instead began “making repetitive movements with her hands” such as clapping, says Coenraads, whose family lived in Virginia at the time.

“I was desperate for a diagnosis,” Coenraads says. But when she finally got one, when Chelsea was 2, “it was a double gut punch.” Not only did Chelsea have Rett syndrome, an untreatable neurological disorder, but scientists knew little about the condition. They understood it primarily affected girls and was likely due to a mutation, but they hadn’t identified the genetic culprit.

That was in 1998. Today, Coenraads and her husband are still caring for Chelsea, now 28, who is unable to speak, walk, or use her hands.

Source: Edit (not Kill) the Messenger | Science