Figure 4.

Motor Neuron Transduction in an Adult NHP Toxicology Study Utilizing ICM Injection

Adult rhesus macaques were administered 1.35 × 10¹³, 4.5 × 10¹², or 1.5 × 10¹² GC (n = 9 per dose) of an AAVhu68 vector expressing human SMN in a volume of 1 mL via ICM injection. Control animals (n = 3) were treated with vehicle. Animals were necropsied 14, 90, or 180 days after vector administration. We quantified the percentage of ChAT⁺ motor neurons expressing human SMN in spinal cord sections from cervical, thoracic, and lumbar levels using ISH. Error bars = SEM.