Figure 3.

scAAV9-CB-IGF1 biodistribution in pigs. A GLP lot of scAAV9-CB-IGF1 was injected intraparenchymally into the cervical cord of two pigs (20 injections, 2.5 × 10¹¹ vg/inj.) or IT via a catheter (500 μL per spinal cord region [cervical, thoracic, and lumbar], 1.4 × 10¹³ vg/mL) in two pigs. Three weeks later, the animals were euthanized and tissues were collected. Quantitative PCR was performed on the brain, spinal cord, and peripheral tissues to measure vector genomes. Missing bars indicate samples where the vector copy number was below the limit of detection. The highest levels of transduction were observed in the cervical cord of pigs receiving parenchymal injections. IT delivery did not result in greater transduction in the thoracic and lumbar cord compared with pigs receiving cervical injections, even though the total dose was nearly sixfold higher. CSF, cerebrospinal fluid; GLP, Good Laboratory Practice; IT, intrathecally; LN, lymph node; ROI, region of interest; vg, vector genomes.