1. Protein LY6E as a candidate for mediating transport of adeno-associated virus across the human blood-brain barrier
- Author
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Eric Kishel, Anetta Ille, Raoul Bodea, Stacy A. Amico-Ruvio, Hannah Lamont, and Alexander M Ille
- Subjects
0301 basic medicine ,Models, Molecular ,viruses ,Lymphocyte ,Gene Expression ,medicine.disease_cause ,Mice ,0302 clinical medicine ,Antigens, Ly ,Protein Isoforms ,Adeno-associated virus ,Cerebral Cortex ,Gene Transfer Techniques ,Dependovirus ,Cell biology ,Molecular Docking Simulation ,medicine.anatomical_structure ,Neurology ,Transcytosis ,Blood-Brain Barrier ,Antigens, Surface ,Receptors, Virus ,Protein Binding ,Central nervous system ,Genetic Vectors ,Gene delivery ,Biology ,Blood–brain barrier ,GPI-Linked Proteins ,Virus ,Capillary Permeability ,03 medical and health sciences ,Cellular and Molecular Neuroscience ,Virology ,medicine ,Animals ,Humans ,Amino Acid Sequence ,Sequence Homology, Amino Acid ,Mechanism (biology) ,Computational Biology ,Endothelial Cells ,Membrane Proteins ,Biological Transport ,Genetic Therapy ,Macaca mulatta ,030104 developmental biology ,Neurology (clinical) ,Sequence Alignment ,030217 neurology & neurosurgery - Abstract
The blood-brain barrier (BBB) is a major obstacle for the treatment of central nervous system (CNS) disorders. Significant progress has been made in developing adeno-associated virus (AAV) variants with increased ability to cross the BBB in mice. However, these variants are not efficacious in non-human primates. Herein, we employed various bioinformatic techniques to identify lymphocyte antigen-6E (LY6E) as a candidate for mediating transport of AAV across the human BBB based on the previously determined mechanism of transport in mice. Our results provide insight into future discovery and optimization of AAV variants for CNS gene delivery in humans.
- Published
- 2020