Figure 4.
Vector Copy Distribution of AAV5 in SCA3 Knockin Mice
(A) Schematic representation of the routes of administration. Three-month-old mice (n = 3) were injected i.c.v., or in the cisterna magna, or DCN with 2.43 × 1013 gc/mL AAV5-miATXN3_7, AAV5-miATXN3_9, or AAV5-miATXN3_11. 10 μL of AAV5 was injected either i.c.v. or in the cisterna magna, and 2 μL was injected bilaterally in the DCN. The injection sites are depicted in red. All mice were sacrificed 6 weeks after surgeries. (B–D) Vector copy distribution in cortex, cerebellum, and brain stem of mice injected i.c.v. (B), or in the cisterna magna (C) or DCN (D) with AAV5-miATXN3. DNA was isolated from the cortex, cerebellum, and brain stem tissues and qRT-PCR was performed to determine the vector copy distribution. The genome copies per μg of DNA were calculated for each brain region using a standard curve. No genomic copies were detected in untreated mice.