Benchmarking the next generation of brain-penetrant AAV capsids

Adeno-associated viral (AAV) vectors have revolutionized the landscape of in vivo gene therapy, emerging as a leading gene delivery platform for treating a wide array of diseases due to their robust safety profile and high transduction efficiency across diverse target tissues. However, translating this clinical success to neurological disorders has historically been impeded by the brain gateway known as the blood-brain barrier (BBB). While this physiological barrier protects the central nervous system (CNS), it effectively prevents most systemically administered therapeutics, including conventional AAVs, from reaching neural tissues.