Immune Responses to the Transgene Product and Capsid in Rhesus Macaques Injected with Suboccipital AAV9.hIDUA
(A) Serum (left panel) and CSF (right panel) anti-hIDUA immune response measured by ELISA are shown. Results are shown as averages per group with SDs as error bars. Humoral responses were observed in the serum and CSF of all vector-treated animals and were not measurable in the HD IS animals at dilutions of 1:1,000 (serum) or 1:20 (CSF). (B) AAV9 NAb titers in serum (left panel) and CSF (right panel) are shown. (C). Interferon-gamma ELISPOT responses in PBMCs, spleen, and bone marrow toward hIDUA (left panel) and AAV9 capsid (right panel) peptide pools.