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. Author manuscript; available in PMC: 2016 Mar 1.
Published in final edited form as: Ann Neurol. 2015 Feb 9;77(3):399–414. doi: 10.1002/ana.24332

Figure 7. scAAV9-SMN treatment at onset of symptoms partially corrects the electrophysiological and histopathological changes observed in SMA affected animals.

Figure 7

(A) Immunofluorescence analysis of lumbar spinal cord sections shows robust GFP (green) and human SMN (red) expression in ChAT-positive cells (blue) of treated pre-symptomatic and treated symptomatic animals with numerous cells expressing both transgenes. (B) LCM-collected lumbar motoneurons were analyzed for pig or human SMN mRNA levels by using ddPCR. Motoneurons from treated pre-symptomatic and treated symptomatic animals have an 87±4% (N=4) and 69±7% (N=5) reduction in pig SMN levels, respectively. Human SMN mRNA levels relative to pig SMN are significantly increased in motoneurons from treated pre-symptomatic and treated symptomatic animals (44±13% and 72±32%). (C) CMAP responses obtained at PND54 in treated symptomatic animals are significantly improved (17.2±2.3) compared\ to SMA-like affected animals (6.8±1.9). MUNE responses were only partially rescued in the treated symptomatic group (225±41 versus 380±25 in control animals) (D) Histopathology performed on the lumbar spinal cord and ventral roots from treated symptomatic animals show scattered chromatolytic motoneurons and axonal degeneration (E) Motoneuron and motor axon counts show a moderate loss of ventral motoneurons and motor axons in treated symptomatic animals indicating partial rescue of the motor unit in these animals. * P<0.05, ** P<0.01. Scale bars: 40μm (A) and 25μm (D).