This year @nature.com published a gene therapy study showing success in mice and monkeys. It did not mention a little girl died from the treatment. That death is in a new @science.org story on the tragedy: bit.ly/4fCAvkk. Nature has not updated the article. The dead girl's family has complained. 🧪
In vivo base editing of Chd3 rescues behavioural abnormalities in mice - Nature
In vivo base editing of a causative mutation that leads to the neurodevelopmental disorder Snijders Blok–Campeau syndrome restores protein dosage and ameliorates molecular and behavioural deficits in ...
nature.com