Intra-Articular AAV9 α-L-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I

Figure 1

Study schematic. Four weeks after birth and six weeks prior to treatment, the three animals underwent pretreatment biopsy (brown vertical line). Treatment (blue vertical line) took place ten weeks after birth with dosages, vectors, and injection sites as designated. Necropsy took place +2 weeks after treatment (I610) or +52 weeks after treatment (I609 and I615). Green arrow indicates treatment with AAV9-eGFP; red arrow indicates treatment with AAV9-IDUA.