Key result
LY6A was identified as an essential receptor for AAV-PHP.B vectors, with ectopic expression of Ly6a increasing AAV-PHP.eB transduction of human cells by 30-fold.
Why the study?
To guide application across disease models and inspire translational gene therapy vectors for neurological diseases, the study sought to elucidate host factors responsible for CNS tropism of AAV-PHP.B vectors.
Population
13 mouse strains, mouse brain endothelial cells, HEK293T cells, and CHO cells
Comparison
Ly6a disruption or blocking antibodies vs ectopic Ly6a expression
Authors
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LY6A enables AAV-PHP.B CNS tropism in mice; hypothesis-generating for human vector engineering pending clinical validation.
p-value: p=<0.001
LY6A is an essential cellular receptor for AAV-PHP.B vectors, enabling efficient gene delivery across the blood-brain barrier.
Huang et al. (2019) studied Gene therapy delivery across the blood-brain barrier. AAV-PHP.eB and Ly6a expression vs. AAV9 or nonpermissive strains/untransfected cells was evaluated on AAV-PHP.eB binding and transduction (p=<0.001). LY6A was identified as an essential receptor for AAV-PHP.B vectors, with ectopic expression of Ly6a increasing AAV-PHP.eB transduction of human cells by 30-fold.
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