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

Figure 6

Synovial DS levels in the (a) high-dose +2 w, (b) high-dose +52 w, and (c) low-dose +52 w animals. Levels of synovial DS GAG at baseline compared to levels at necropsy. AAV9-IDUA joints in all three animals demonstrate synovial DS reduction, while AAV9-eGFP-treated joints show increases in DS levels in the two +52 w animals. The etiology for the DS reduction in AAV9-eGFP-treated joints in the +2 w-treated animal is unclear as its synovium has no measurable IDUA enzyme.

(a)

(b)

(c)