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. 2022 Dec 1;30(5):443–454. doi: 10.1038/s41434-022-00375-w

Fig. 1. Neonatal ICV injection of AAV-sgSOD1 effectively reduces huSOD1 protein in the CNS and generates high indel rates in the huSOD1 gene.

Fig. 1

a Study design of histological and biochemical analysis on H11Cas9 SOD1G93A mice treated with AAV-sgSOD1 (Cohort #1). b Percentage of remaining huSOD1 protein in cortex (left) and spinal cord (right) of H11Cas9 SOD1G93A mice treated with AAV-sgSOD1 (Cohort #1) as determined by ELISA. c Indel percentage in cortex and spinal cord of H11Cas9 SOD1G93A mice treated with AAV-sgSOD1 (Cohort #1) as determined by amplicon-Seq of transduced nuclei (left) and all nuclei (right). Data are presented as mean with standard deviation (SD) unless otherwise stated. Welch’s t-test with Bonferroni correction is performed unless otherwise stated. *p < 0.05, **p < 0.01, and ***p < 0.001, n.s. not significant. See also Fig. S3.