Table 1 GAA activity restoration and glycogen storage correction in the CNS 12Â 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) |