%A Arotcarena,Marie-Laure %A Dovero,Sandra %A Biendon,Nathalie %A Dutheil,Nathalie %A Planche,Vincent %A Bezard,Erwan %A Dehay,Benjamin %D 2021 %J Frontiers in Bioengineering and Biotechnology %C %F %G English %K adeno-associated virus,Intrathecal injection,CNS,non-human primate,animal model %Q %R 10.3389/fbioe.2021.762209 %W %L %M %P %7 %8 2021-November-15 %9 Original Research %# %! Intrathecal delivery for brain transduction in non-human primates %* %< %T Pilot Study Assessing the Impact of Intrathecal Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques %U https://www.frontiersin.org/articles/10.3389/fbioe.2021.762209 %V 9 %0 JOURNAL ARTICLE %@ 2296-4185 %X 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.