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

Figure 3

(a) IDUA enzyme activity measurements from homogenates from wild-type, MPS-I untreated, and study animals. Data were generated from at least three independent experiments and shown as . (b) Schematic of PCR primers utilized to detect the IDUA transgene (389 base pairs spanning cDNA between exon 10 and exon 14) and the native genomic IDUA gene (227 bp covering the entirety of exon 7). (c) Transgene and genomic IDUA PCR of liver homogenates. The presence of the native IDUA gene is identified in all animals, but only the high-dose +2 w dog demonstrates the presence of IDUA transgene and appreciable levels of IDUA enzyme in liver. The two +52 w animals had neither measurable liver IDUA enzyme activity nor IDUA transgene.

(a)

(b)

(c)