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Abstract
To optimize gene delivery to myelinating Schwann cells we compared clinically relevant AAV serotypes and injection routes. AAV9 and AAVrh10 vectors expressing either EGFP or the neuropathy-associated gene GJB1/Connexin32 (Cx32) under a myelin specific promoter were injected intrathecally or intravenously in wild type and Gjb1-null mice, respectively. Vector biodistribution in lumbar roots and sciatic nerves was higher in AAVrh10 injected mice while EGFP and Cx32 expression rates and levels were similar between the two serotypes. A gradient of biodistribution away from the injection site was seen with both intrathecal and intravenous delivery, while similar expression rates were achieved despite higher vector amounts injected intravenously. Quantified immune cells in relevant tissues were similar to non-injected littermates. Overall, AAV9 and AAVrh10 efficiently transduce Schwann cells throughout the peripheral nervous system with both clinically relevant routes of administration, although AAV9 and intrathecal injection may offer a more efficient approach for treating demyelinating neuropathies.
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Details
1 The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Neuroscience Department, Nicosia, Cyprus (GRID:grid.417705.0) (ISNI:0000 0004 0609 0940)
2 The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Molecular Virology Department, Nicosia, Cyprus (GRID:grid.417705.0) (ISNI:0000 0004 0609 0940)
3 Institute of Neurosciences, Department of Biochemistry and Molecular Biology, Barcelona, Spain (GRID:grid.417705.0); Unitat Mixta UAB-VHIR, Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain (GRID:grid.430994.3) (ISNI:0000 0004 1763 0287)
4 Institute of Neurosciences, Department of Biochemistry and Molecular Biology, Barcelona, Spain (GRID:grid.417705.0); Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Bellaterra, Spain (GRID:grid.418264.d) (ISNI:0000 0004 1762 4012); Unitat Mixta UAB-VHIR, Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain (GRID:grid.430994.3) (ISNI:0000 0004 1763 0287)
5 The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Neuroscience Department, Nicosia, Cyprus (GRID:grid.417705.0) (ISNI:0000 0004 0609 0940); The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Center for Neuromuscular Diseases, Nicosia, Cyprus (GRID:grid.417705.0) (ISNI:0000 0004 0609 0940)