Cell reportsDependovirusHumansAnimalsBrainMice
AAVs targeting human carbonic anhydrase IV enhance gene delivery to the brain.
Changfan Lin, Xinhong Chen, Jonathan D Hoang, Fiona Ristic, Yujie Fan, Seongmin Jang, Jin Hyung Alex Chung, Erin E Sullivan, Tomasz Gawda, Bill Kavvathas, Irene Tran, Yitong Li, Andrew D Steele, Timothy F Shay, Viviana Gradinaru
Published: 202510.1016/j.celrep.2025.116419
Abstract
Gene therapies using natural adeno-associated virus (AAV) serotypes have restricted applications, particularly in the brain, due to their poor targeting and resulting safety concerns. Directed evolution has identified brain-enhanced engineered AAV ca…
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