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. 2021 Oct 15;11:20513. doi: 10.1038/s41598-021-99979-2

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© The Author(s) 2021

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.

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Expression of GFP and ARSA in the brains of treated MLD mice. AAV9/ARSA (encoding ARSA and GFP) were intrathecally injected via suboccipital puncture into 1-year-old MLD mice. (a) representative images of immunostaining for GFP and ARSA in sagittal brain and spinal cord sections taken 3 months after injection of AAV9/ARSA. Boxes indicate the approximate regions from which the higher magnification images shown in b were acquired. (b) 1, caudate putamen (striatum); 2, inferior colliculus; 3, cerebellum; 4, ventral horn of the cervical spinal cord. Bar: 200 μM. Panels on the right highlight the observed co-localization. (c) ARSA levels in the indicated brain regions measured with ELISAs in MLD mice treated at 6 week (n = 4) and 1 year (n = 5) of age. (d) droplet digital PCR to detect the copy numbers of AAV vector in MLD mice treated at 6 week (n = 4) and 1 year (n = 5) and of age. FB, forebrain; HB, hindbrain; SC, spinal cord; L, liver; ARSA, human arylsulfatase A.