Figure 4
From: A novel mucopolysaccharidosis type II mouse model with an iduronate-2-sulfatase-P88L mutation
The effect of nuclease-mediated correction of IDS enzyme activity. (A) Timeline of the experimental protocol. Prior to the experiments, the mice were weighed and randomized based on body weight. The mice were grouped as (1) a control group with wild-type mice treated with PBS; (2) an MPS II disease group with Ids-P88L MPS II mouse model treated with PBS; and (3) a treated group with an Ids-P88L MPS II mouse model treated with a mixture of Cas9 nuclease, gRNA, and ssODN for template DNA. At day 7, the blood was collected by retro-orbital plexus and spotted onto filter paper, dried at room temperature overnight, and stored at -20 °C prior to use. (B) The IDS enzyme activity of the nuclease-treated Ids-P88L MPS II mouse model. The enzyme activity was quantified using LC–MS/MS as described. Open circle, control group (n = 4); closed circle, Ids-P88L MPS II mouse model (n = 4); closed triangle, treated group (n = 4). A bar indicates the mean value. *P < 0.05. Inset, enzyme activity of Ids-P88L MPS II mouse model treated with PBS and nuclease-were magnified.
