dc.contributor
Institut Català de la Salut
dc.contributor
[Kagiava A, Sargiannidou I] Neuroscience Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, 1683 Nicosia, Cyprus. [Richter J, Tryfonos C, Christodoulou C] Molecular Virology Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Nicosia, Cyprus. [Leal-Julià M] Department of Biochemistry and Molecular Biology, Institute of Neurosciences, Barcelona, Spain. Unitat Mixta UAB-VHIR, Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain. [Bosch A] Department of Biochemistry and Molecular Biology, Institute of Neurosciences, Barcelona, Spain. Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Bellaterra, Spain. Unitat Mixta UAB-VHIR, Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain
dc.contributor
Vall d'Hebron Barcelona Hospital Campus
dc.contributor.author
Richter, J.
dc.contributor.author
Tryfonos, C.
dc.contributor.author
Leal Julià, Marc
dc.contributor.author
Christodoulou, C.
dc.contributor.author
Bosch Merino, Assumpció
dc.contributor.author
Kagiava, Alexia
dc.contributor.author
Sargiannidou, Irene
dc.date.accessioned
2025-10-24T08:53:40Z
dc.date.available
2025-10-24T08:53:40Z
dc.date.issued
2022-05-25T05:59:12Z
dc.date.issued
2022-05-25T05:59:12Z
dc.date.issued
2021-11-02
dc.identifier
Kagiava A, Richter J, Tryfonos C, Leal-Julià M, Sargiannidou I, Christodoulou C, et al. Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery. Sci Rep. 2021 Dec 2;11:23358.
dc.identifier
https://hdl.handle.net/11351/7576
dc.identifier
10.1038/s41598-021-02694-1
dc.identifier
000728529200081
dc.identifier.uri
http://hdl.handle.net/11351/7576
dc.description.abstract
Diseases of the nervous system; Myelin biology and repair; Peripheral nervous system
dc.description.abstract
Enfermedades del sistema nervioso; Biología y reparación de la mielina; Sistema nervioso periférico
dc.description.abstract
Malalties del sistema nerviós; Biologia i reparació de la mielina; Sistema nerviós perifèric
dc.description.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.
dc.description.abstract
Generalitat de Catalunya, 2019FI_B2 00061, 2019FI_B2 00061, Fundació la Marató de TV3, 201607.10, Muscular Dystrophy Association, 603003.
dc.format
application/pdf
dc.format
application/pdf
dc.publisher
Nature Research
dc.relation
Scientific Reports;11
dc.relation
https://doi.org/10.1038/s41598-021-02694-1
dc.rights
Attribution 4.0 International
dc.rights
http://creativecommons.org/licenses/by/4.0/
dc.rights
info:eu-repo/semantics/openAccess
dc.subject
Mielina - Metabolisme
dc.subject
Sistema nerviós - Malalties
dc.subject
ANATOMY::Nervous System::Nervous System::Peripheral Nervous System::Peripheral Nerves::Schwann Cells
dc.subject
Other subheadings::Other subheadings::Other subheadings::/metabolism
dc.subject
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Drug Therapy::Drug Administration Routes::Injections::Injections, Spinal
dc.subject
DISEASES::Nervous System Diseases::Demyelinating Diseases
dc.subject
ANATOMÍA::sistema nervioso::sistema nervioso::sistema nervioso periférico::nervios periféricos::células de Schwann
dc.subject
Otros calificadores::Otros calificadores::Otros calificadores::/metabolismo
dc.subject
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::farmacoterapia::vías de administración de medicamentos::inyecciones::inyecciones espinales
dc.subject
ENFERMEDADES::enfermedades del sistema nervioso::enfermedades desmielinizantes
dc.title
Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery
dc.type
info:eu-repo/semantics/article
dc.type
info:eu-repo/semantics/publishedVersion
