What we can learn from the first personalized CRISPR-treated baby to tackle genetic brain disorders.
Neuron, 113(22): 3697-3702 PMID 41232532 DOI 10.1016/j.neuron.2025.10.023
Abstract
The landmark report on personalized CRISPR genome editing to treat an infant (baby KJ) with a life-threatening liver disease sparked widespread attention,1 ushering in a new era of precision genetic intervention. This piece discusses the key challenges and opportunities in translating this milestone into treatments for genetic brain disorders.