Fig. 2.

Intrathecal gene therapy provides long-term neurologic correction. Pompe mice (−/−) were injected at 1 month in the cisterna magna with 10¹¹ vg of AAVrh10-CAG-hGAA (n = 12) or AAV9-CAG-hGAA (n = 12) or PBS (n = 11) and their neurologic function tests results were compared to those of wild-type (WT) mice of the same genetic background (b6;129) injected with PBS (n = 15). a Hindleg clasping reflex score from 0 (normal hindleg placement) to 4 (permanent abnormal retraction of both hindlegs). Note the rapid and progressive neurological deficit observed in mock-treated Pompe mice (−/−) PBS only (Linear mixed effects, time effect: P < 0.0001). b Coordination evaluation by accelerating rotarod test (4 to 40 rotations per minute in 3 min). c The latency between the first and the fifth peak of the brainstem auditory response representing a fully restored nerve conduction velocity within the auditory brainstem in treated animals (Linear mixed effects, group effect: P < 0,0001)