Brain-wide genome editing via a single systemic dose of modified adeno-associated virus variants that cross the blood-brain barrier may represent a promising new approach for the development of disease-modifying treatments for familial Alzheimer's disease. This strategy could also be applicable to other central nervous system (CNS) disorders, according to a proof-of-concept (PoC) study led by researchers at The Hong Kong University of Science and Technology (HKUST).