AROTCARENA, Marie Laure; DOVERO, Sandra; BIENDON, Nathalie; DUTHEIL, Nathalie; PLANCHE, Vincent; BEZARD, Erwan; DEHAY, Benjamin

doi:10.3389/fbioe.2021.762209

Institut des Maladies Neurodégénératives [Bordeaux] [IMN]

DOVERO, Sandra
Institut des Maladies Neurodégénératives [Bordeaux] [IMN]

BIENDON, Nathalie
Institut des Maladies Neurodégénératives [Bordeaux] [IMN]

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Article de revue

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Frontiers in Bioengineering and Biotechnology. 2021-11-15, vol. 9

Résumé en anglais

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.< Réduire

Mots clés en anglais

Adeno-associated virus

Intrathecal injection

CNS

Non-human primate

Animal model

URI

https://oskar-bordeaux.fr/handle/20.500.12278/148369

DOI

http://dx.doi.org/10.3389/fbioe.2021.762209

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Institut des Maladies Neurodégénératives (IMN) - UMR 5293

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Pilot Study Assessing the Impact of Intrathecal Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques

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