AAVs targeting human carbonic anhydrase IV enhance gene delivery to the brain
Changfan Lin, Xinhong Chen, Jonathan D. Hoang, Fiona Ristic et autres
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 capsids for various model organisms, but inter-species differences challenge their translation. …
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8 citations
Cell Reports