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. 2015 Jan 6;23(2):270–277. doi: 10.1038/mt.2014.209

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Copyright © 2015 American Society of Gene & Cell Therapy

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scAAV6-mediated knockdown of PTEN ameliorates neuromuscular junction (NMJ) pathology in SMA mice. (a) Representative confocal micrographs showing widespread NMJ pathology in the caudal band of the levator auris longus muscle (LAL) in SMNΔ7 mice at postnatal day 10 that received intramuscular injection of scrambled siPTEN (AAV6-ssiPTEN) at postnatal day 1 (left panel) and reduced pathology in mice that received intramuscular injection of siPTEN (AAV6-siPTEN). Antibodies against neurofilament proteins were used to label motor axon collaterals (green), and tetramethylrhodamine-conjugated α-bungarotoxin was used to label acetylcholine receptors at the motor endplate (red). More motor endplates appeared to be innervated by overlying motor nerve terminals in the AAV6-siPTEN–treated muscle. (b) Bar chart showing the extent of NMJ denervation (mean ± SEM) in the affected caudal band, and unaffected rostral band, of the LAL muscle in treated (siRNA) and untreated (ssiRNA) mice. This analysis revealed significantly more innervated NMJs in the caudal band of treated mice (*P < 0.05, n = 4 mice per group, Student's t-test).