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Guo Y, Wang D, Qiao T, Yang C, Su Q, Gao G, Xu Z (2016) A Single Injection of Recombinant Adeno-Associated Virus into the Lumbar Cistern Delivers Transgene Expression Throughout the Whole Spinal Cord. Mol Neurobiol 53:3235-3248.

Wang H, Ghosh A, Baigude H, Yang C-s, Qiu L, Xia X, Zhou H, Rana TM, Xu Z (2008) Therapeutic Gene Silencing Delivered by a Chemically Modified Small Interfering RNA against Mutant SOD1 Slows Amyotrophic Lateral Sclerosis Progression. J Biol Chem 283:15845-15852.

Wang H, Yang B, Qiu L, Yang C, Kramer J, Su Q, Guo Y, Brown RH, Jr., Gao G, Xu Z (2014) Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Genet 23:668-681.

Wu R, Wang H, Xia X, Zhou H, Liu C, Castro M, Xu Z (2009) Nerve Injection of Viral Vectors Efficiently Transfers Transgenes into Motor Neurons and Delivers RNAi Therapy Against ALS. Antioxidants & Redox Signaling 11:1523-1534.

Xia X, Zhou H, Huang Y, Xu Z (2006) Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo. Neurobiol Dis 23:578-586.

Xu Z, Jung C, Higgins C, Levine J, Kong J (2004) Mitochondrial degeneration in amyotrophic lateral sclerosis. J Bioenerg Biomembr 36:395-399.

Yang C, Danielson EW, Qiao T, Metterville J, Brown RH, Jr., Landers JE, Xu Z (2016) Mutant PFN1 causes ALS phenotypes and progressive motor neuron degeneration in mice by a gain of toxicity. Proc Natl Acad Sci U S A.

Yang C, Wang H, Qiao T, Yang B, Aliaga L, Qiu L, Tan W, Salameh J, McKenna-Yasek DM, Smith T, Peng L, Moore MJ, Brown RH, Jr., Cai H, Xu Z (2014) Partial loss of TDP-43 function causes phenotypes of amyotrophic lateral sclerosis. Proc Natl Acad Sci U S A 111:E1121-1129.