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. 2022 Jun 10;33(11-12):579–597. doi: 10.1089/hum.2022.037

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© Annie Tanenhaus et al. 2022; Published by Mary Ann Liebert, Inc.

This Open Access article is distributed under the terms of the Creative Commons License [CC-BY] (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Figure 5. — Unilateral ICV delivery of AAV9-RE^GABA-eTF^SCN1A to NHPs leads to widespread vector biodistribution and increased eTF^SCN1A mRNA transcript levels in the brain with low off-target vector expression in peripheral tissues of NHPs. (A) Study design. AAV9-RE^GABA-eTF^SCN1A (4.8–8.0 E13 vg/animal) or vehicle alone (PBS) was administered by unilateral ICV injection to four juvenile cynomolgus macaques (3M and 1F). All animals were sacrificed 28 ± 2 days after injection. Biodistribution of AAV9-RE^GABA-eTF^SCN1A vector copies and expression of transgene mRNA were measured in target neuronal and peripheral tissues using ddPCR (B–E). (B) AAV9-RE^GABA-eTF^SCN1A vector biodistribution in brain regions. (C) eTF^SCN1A mRNA expression levels in brain regions. (D) AAV9-RE^GABA-eTF^SCN1A vector biodistribution and (E) eTF^SCN1A mRNA expression levels in peripheral tissues. (F) VCN:RNA ratios for brain and peripheral tissues (N = 4, mean values; error bars indicate SD). Starred organs (*) indicate VCN or RNA levels below the limit of detection of the assay. DRG, dorsal root ganglia; NAb, neutralizing antibodies; NHP, nonhuman primate; SC, spinal cord.