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Table 1 GAA activity restoration and glycogen storage correction in the CNS 12 months after intrathecal AAV-CAG-hGAA therapy

From: Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease

Treatment Tissue Mean GAA activity (nmol/h/mg prot) Mean GAA activity (% WT) Mean glycogen correction relative to mock-treated (% reduction)
AAVrh10 n = 4 Rostral brain 1.8 (±0.6) 6.1 (±2.2) 84.3 (±6.5)
Brainstem 35 (±21.9) 37.8 (±23.6) 83 (±15.4)
Proximal spinal cord 10.4 (±1.7) 13.5 (±2.2) 91.9 (±5.3)
Distal spinal cord 2.8 (±0.7) 5 (±1.2) 81.1 (±11.4)
AAV9 n = 4 Rostral brain 2.4 (±1.1) 8.3 (±3.9) 82.5 (±10.8)
Brainstem 31.6 (±20.8) 34 (±22.4) 81.6 (±12.1)
Proximal spinal cord 7.6 (±1.7) 9.9 (±2.3) 88.9 (±6.1)
Distal spinal cord 5.1 (±0.3) 9 (±0.5) 78.3 (±8.5)