Pilot Study Assessing the Impact of Intrathecal Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques
| dc.rights.license | open | en_US |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | AROTCARENA, Marie Laure | |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | DOVERO, Sandra | |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | BIENDON, Nathalie | |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | DUTHEIL, Nathalie | |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | PLANCHE, Vincent | |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | BEZARD, Erwan | |
| hal.structure.identifier | Institut des Maladies Neurodégénératives [Bordeaux] [IMN] | |
| dc.contributor.author | DEHAY, Benjamin | |
| dc.date.accessioned | 2022-10-10T09:01:59Z | |
| dc.date.available | 2022-10-10T09:01:59Z | |
| dc.date.issued | 2021-11-15 | |
| dc.identifier.issn | 2296-4185 | en_US |
| dc.identifier.uri | oai:crossref.org:10.3389/fbioe.2021.762209 | |
| dc.identifier.uri | https://oskar-bordeaux.fr/handle/20.500.12278/148369 | |
| dc.description.abstractEn | Adeno-associated virus (AAV) vectors are increasingly used as an effective and safe approach to deliver genetic material to the central nervous system (CNS). The AAV9-derived variants, AAV-PHP. B and AAV-PHP.eB, reportedly broadly transduce cells throughout the CNS compared to the original serotype 9, AAV9. As non-human primate data are scarce, we here evaluated the CNS transduction efficiencies after lumbar intrathecal bolus delivery of identical doses of either AAV-PHP. B:CAG-EGFP or AAV-PHP. eB:CAG-EGFP in rhesus macaque monkeys. AAV-PHP.eB achieved a more efficient and widespread CNS transduction compared to AAV-PHP.B. We report a strong neuronal and oligodendroglial tropism for both variants in the putamen and in the hippocampus. This proof-of-concept experiment highlights the potential value of intrathecal infusions of AAV-PHP.eB to distribute genetic material in the CNS with cell-type specificity and introduces a new opportunity to model brain diseases in rhesus macaque monkeys and further develop gene therapies targeting the CNS in humans. | |
| dc.language.iso | EN | en_US |
| dc.rights | Attribution 3.0 United States | * |
| dc.rights.uri | http://creativecommons.org/licenses/by/3.0/us/ | * |
| dc.source | crossref | |
| dc.subject.en | Adeno-associated virus | |
| dc.subject.en | Intrathecal injection | |
| dc.subject.en | CNS | |
| dc.subject.en | Non-human primate | |
| dc.subject.en | Animal model | |
| dc.title.en | Pilot Study Assessing the Impact of Intrathecal Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques | |
| dc.type | Article de revue | en_US |
| dc.identifier.doi | 10.3389/fbioe.2021.762209 | en_US |
| dc.subject.hal | Sciences du Vivant [q-bio] | en_US |
| dc.identifier.pubmed | 34869273 | en_US |
| bordeaux.journal | Frontiers in Bioengineering and Biotechnology | en_US |
| bordeaux.volume | 9 | en_US |
| bordeaux.hal.laboratories | Institut des Maladies Neurodégénératives (IMN) - UMR 5293 | en_US |
| bordeaux.institution | Université de Bordeaux | en_US |
| bordeaux.institution | CNRS | en_US |
| bordeaux.peerReviewed | oui | en_US |
| bordeaux.inpress | non | en_US |
| bordeaux.identifier.funderID | Ministère de l'Enseignement supérieur, de la Recherche et de l'Innovation | en_US |
| bordeaux.identifier.funderID | Association France Parkinson | en_US |
| bordeaux.identifier.funderID | Université de Bordeaux | en_US |
| bordeaux.identifier.funderID | Centre National de la Recherche Scientifique | en_US |
| bordeaux.import.source | dissemin | |
| hal.identifier | inserm-03429973 | |
| hal.version | 1 | |
| hal.export | false | |
| workflow.import.source | dissemin | |
| dc.rights.cc | Pas de Licence CC | en_US |
| bordeaux.COinS | ctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.jtitle=Frontiers%20in%20Bioengineering%20and%20Biotechnology&rft.date=2021-11-15&rft.volume=9&rft.eissn=2296-4185&rft.issn=2296-4185&rft.au=AROTCARENA,%20Marie%20Laure&DOVERO,%20Sandra&BIENDON,%20Nathalie&DUTHEIL,%20Nathalie&PLANCHE,%20Vincent&rft.genre=article |
