Figure 1.

Motor Neuron Transduction following AAVhu68 Administration via Intra-cisterna Magna (ICM) Injection or Lumbar Puncture (LP) to Adult and Juvenile NHPs

Adult (3- to 6-year-old) and juvenile (12- to 14-month-old) rhesus macaques were administered 3 × 10¹³ GC of an AAVhu68 vector expressing human SMN from a chicken beta-actin promoter. The vector was injected into the cisterna magna (ICM) in a volume of 1 mL or via LP in a volume of 5 mL. Animals were necropsied 28 days after vector administration. We performed in situ hybridization on spinal cord sections using probes for the human SMN transgene and choline acetyltransferase (A). We quantified the percentage of ChAT⁺ motor neurons expressing human SMN in spinal cord sections from cervical, thoracic, and lumbar levels (B). Error bars = standard error of the mean (SEM).