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Updated: May 11, 2026

Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling
Published on: April 2, 2012
Complete neutralizing antibody evasion by serodivergent non-mammalian AAVs enables gene therapy redosing
Ezra J Loeb1, Sophia A Fergione2, Vivian Yudistyra2
1Department of Biomedical Engineering, Duke University, Durham, NC, USA.
Abstract:
The use of adeno-associated virus (AAV) as a gene therapy vector is significantly limited by pre-existing immunity. The high seroprevalence and broad antigenic cross-reactivity of primate-derived AAVs restrict patient eligibility and preclude therapeutic redosing. Here, we harness the phylogenetic diversity of non-mammalian dependoparvoviruses to engineer serologically distinct AAV capsids for immune evasion. A barcoded screen of divergent Dependoparvovirus isolates identifies AAV.div3A, a chimeric capsid with robust transduction, zero antigenic cross-reactivity, and undetectable seroprevalence. Derived from a phylogenetically distant Muscovy duck isolate, AAV.div3A fully evades neutralization in mice, even after passive immunization with NAb+ human serum or following initial vector dosing. Further engineering yields AAV.div3A-M1, a myotropic, liver-detargeted capsid with enhanced cardiac and diaphragm transduction. In a Pompe disease model, redosing with AAV.div3A or div3A-M1 significantly increases therapeutic GAA levels. Overall, our work leverages untapped dependoparvoviral diversity to overcome pre-existing and vector-induced immunity, enabling expansion of patient eligibility and effective redosing.
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