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- Kong, J-M and Xu, Z: Massive mitochondrial degeneration in motor neurons triggers the onset of amyotrophic lateral sclerosis in mice expressing a mutant SOD1. J Neurosci, 18: 3241-3250 (1998).
- Borchelt DR, Wong PC, Becher MW, Pardo CA, Lee MK, Xu Z, Thinakaran G, Jenkins NA, Copeland NG, Sisodia SS, Cleveland DW, Price DC, and Hoffman PN: Axonal transport of mutant superoxide dismutase 1 and focal axonal abnormalities in the proximal axons of transgenic mice. Neurobiol Dis 5: 27-35 (1998).
- Bogdanov MB, Ramos LE, Xu Z, and Beal MF: Elevated hydroxyl radical generation in vivo in an animal model of amyotrophic lateral sclerosis. J Neurochem 71: 1321-24 (1998).
- Kong J-M and Xu Z: Peripheral Axotomy Slows Motor Neuron Degeneration in a Transgenic Mouse Line Expressing Mutant SOD1 G93A. J Comp Neurol 412: 373-380 (1999).
- Levine J, Kong J-M and Xu Z: Astrocytes interact intimately with degenerating motor neurons in mouse amyotrophic lateral sclerosis (ALS). Glia 28: 215-224 (1999).
- Kong J-M and Xu Z: Overexpression of neurofilament subunit NF-L and NF-H extends survival of a mouse model for ALS. Neurosci Let 281: 72-4 (2000).
- Xu Z and Tung V: Overexpression of neurofilament subunit M accelerates neurofilament transport. Brain Res 866: 326-32 (2000).
- Xu Z: Mechanism and treatment of motoneuron degeneration in ALS: what have SOD1 mutants told us? ALS and Other Motor Neuron Diseases 1: 225-34 (2000).
- Jung C and Xu Z: Measuring Quantity and Activity of Mitochondrial Electron Transport Chain (ETC) Complexes in Nervous Tissues Using BN-PAGE. Anal Biochem 286:214-23 (2000).
- Xu ZS and Tung V: Temporal and spatial variations in slow axonal transport velocity along peripheral motoneuron axons. Neuroscience 102: 193-200 (2001).
- Jung C, Rong Y, Doctrow S, Baudry M, Malfroy B and Xu ZS: Prolonged survival of ALS mice treated with synthetic superoxide dismutase/catalase mimetics. Neurosci Let 304: 157-60 (2001).
- Jung C, Higgins CJM and Xu ZS: A quantitative histochemical assay for activities of mitochondrial electron transport chain complexes in mouse spinal cord sections. J Neurosci Meth 114: 165-72 (2002).
- Higgins CJM, Jung C, Ding HL and Xu ZS: Mutant Cu, Zn superoxide dismutase that causes motoneuron degeneration is present in mitochondria in the central nervous system. J Neurosci RC215:1-6 (2002).
- Zhang Z, Casey D, Julien J-P and Xu ZS: Neurofilament subunit L (NF-L) is required for normal development of motoneuron dendrite. J Comp Neurol 450:144-52 (2002).
- Jung C, Higgins CJM and Xu ZS: Mitochondrial electron transport chain complex dysfunction in a transgenic mouse model for amyotrophic lateral sclerosis. J Neurochem 83: 535-45 (2002).
- Ding HL, Schwarz DS, Keene A, Affar E, Fenton L, Xugang Xia, Shi Y, Zamore PD and Xu ZS: Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis. Aging Cell 2: 209-217 (2003).
- Higgins CJM, Jung, CW, Xu ZS: ALS-associated mutant SOD1G93A causes mitochondrial vacuolation by expansion of the intermembrane space and involvement of peroxisomes. BMC Neuroscience 4: 16 (2003).
- Xia XG, Zhou HX, Ding HL, Affar EB, Shi Y, Xu ZS: An enhanced U6 promoter for synthesis of small hairpin RNA. Nuc Acids Res, e100 (2003).
- Schwarz DS, Hutvágner G, Du TT, Xu ZS, Aronin N, Zamore PD: Asymmetry in the assembly of the RNAi enzyme complex. Cell 115: 199-208 (2003).
- Cozzolino M, Ferraro E, Ferri E, Rigamonti D, Quondamatteo F, Ding H, Xu ZS, Ferrari F, Angelini DF, Rotilio G, Cattaneo E, Carrı´ MT and Cecconi F: Apoptosome inactivation rescues proneural and neural cells from neurodegeneration. Cell Death and Differentiation 11: 1179-91 (2004).
- Xu Z: Mechanism and treatment of motoneuron degeneration in ALS: what have SOD1 mutants told us? ALS and Other Motor Neuron Diseases 1: 225-34 (2000).
- Xia XG, Zhou H, Zhou S, Yu Y, Wu R and Xu ZS (2005) An RNAi strategy for treatment of amyotrophic lateral sclerosis caused by mutant Cu, Zn superoxide dismutase. J Neurochem 92:362-367 (2005).
- Tummala H, Jung C, Tiwari A, Higgins CMJ, Hayward LJ, Xu Z (2005) Inhibition of chaperone activity is a shared property of several Cu, Zn superoxide dismutase mutants that cause amyotrophic lateral sclerosis. J Biol Chem 280:17725-17731 (2005).
- Zhou H, Xia XG, Xu Z (2005) An RNA polymerase II construct synthesizes short-hairpin RNA with a quantitative indicator and mediates highly efficient RNAi. Nucl Acids Res 33:e62 (pp1-8) (2005).
- Tiwari A, Xu Z, Hayward LJ (2005) Aberrantly increased hydrophobicity shared by mutants of Cu/Zn superoxide dismutase in familial amyotrophic lateral sclerosis. J Biol Chem 280: 29771-9 (2005).
- Xu ZS and Kong JM (2005) Role of mitochondrial damage in ALS. In “Amyotrophic Lateral Sclerosis”, edited by Mitsumoto et al., published by Taylor & Francis, London. (2005).
- Manfredi G, Xu Z (2005) Mitochondrial dysfunction and its role in motor neuron degeneration in ALS. Mitochondrion 5:77-87 (2005).
- Xu Z, Xia XG (2005) RNAi therapy: Dominant disease gene gets silenced. Gene Ther 12:1159-60 (2005).
- Xia XG, Zhou H, Xu ZS (2005) Promises and Challenges in Developing RNAi as a Research Tool and Therapy for Neurodegenerative Diseases. Neurodeg Dis 2: 220-31 (2005).
- Schonhoff CM, Matsuoka M, Tummala H, Johnson MA, Estevéz AG, Wu R, Kamaid A, Ricart KC, Hashimoto Y, Gaston B, Macdonald TL, Xu Z, Mannick JB (2006) S-nitrosothiol depletion in amyotrophic lateral sclerosis. PNAS 103: 2404-2409 (2006).
- Xu Gang Xia, Hongxia Zhou, Enrique Samper, Simon Melov and Zuoshang Xu (2006) Pol II-expressed shRNA knocks down Sod2 gene expression and causes phenotypes of the gene knockout in mice. PloS Genetics 2: e10 (2006).
- Hitoshi Kikuchi, Gabriele Almer, Satoshi Yamashita, Christelle Gue´gan, Makiko Nagai, Zuoshang Xu, Alexander A. Sosunov, Guy M. McKhann II, and Serge Przedborski (2006) Spinal cord endoplasmic reticulum stress associated with a microsomal accumulation of mutant superoxide dismutase-1 in an ALS model. PNAS 103: 6025-30 (2006).
- Xu-Gang Xia, Hongxia Zhou, and Zuoshang Xu (2006) Multiple shRNAs expressed by an inducible pol II promoter can knock down the expression of multiple target genes. BioTechniques 41: 64-68 (2006).
- Xugang Xia, Hongxia Zhou, Yong Huang and Zuoshang Xu (2006) Allele-specific RNAi Selectively Silences Mutant SOD1 and Achieves Significant Therapeutic Benefit in vivo. Neurobiol Dis 23: 578-586 (2006).
- Dianne S. Schwarz*, Hongliu Ding, Lori Kennington, Jess T. Moore, Janelle Schelter, Peter S. Linsley, Neil Aronin, Zuoshang Xu*, and Phillip D. Zamore*. Designing Functionally Asymmetric siRNA with Single Nucleotide Discrimination. PLoS Genetics 2(9): e140. *corresponding authors (2006).
- Xu-Gang Xia, Hongxia Zhou and Zuoshang Xu (2006) Transgenic RNAi: Accelerating and Expanding Reverse Genetics in Mammals. Transgenic Res 15: 271-5 (2006).
- Zuoshang Xu and Xu Gang Xia (2006) Reverse Genetics in Mammals Using RNAi. Cellscience 3(1): (2006).
- Zhou H, Falkenburger BH, Schulz JB, Tieu K, Xu Z, Xia XG. Silencing of the Pink1 gene expression by conditional RNAi does not induce dopaminergic neuron death in mice. Int J Biol Sci 3:242-250. *corresponding authors (2007).
- Zou T, Ilangovan R, Yu F, Xu Z, Zhou J. SMN protects cells against mutant SOD1 toxicity by increasing chaperone activity. Biochem Biophys Res Comm 364:850-855 (2007).
- Wang H, Ghosh A, Baigude H, Yang CS, Qiu L, Xia X, Zhou H, Rana TM and Xu Z. Therapeutic gene silencing delivered by a chemically modified siRNA against mutant SOD1 slows ALS progression. J Biol Chem 283: 15845-15852 (featured as “paper of the week”) (2008).
- Linghua Qiu, Hongyan Wang, Xugang Xia, Hongxia Zhou and Zuoshang Xu. A construct with fluorescent indicators for conditional expression of miRNA. BMC Biotechnology 8:77 (designated as “highly accessed”) (2008).
- Rui Wu, Hongyan Wang, Xugang Xia, Hongxia Zhou, Chunyan Liu, Maria Castro and Zuoshang Xu. Nerve injection of viral vectors efficiently transfers transgenes into motor neurons and delivers RNAi therapy against ALS. Antioxid Redox Signal 11: 1523-1534 (2009).
- Qiu L, Xu Z Transgenic RNAi—a fast and low-cost approach to reverse genetics in mammals. In: Regulation of Gene Expression by Small RNAs (Gaur RK, Rossi JJ, eds), pp 317-331. Boca Raton: CRC Press (2009).
- Chunxing Yang, Weijia Tan, Catheryne Whittle, Linghua Qiu, Lucheng Cao, Schahram Akbarian and Zuoshang Xu. The C-terminal TDP-43 fragments have a high aggregation propensity and harm neurons by a dominant-negative mechanism. PLoS One 5: e15878 (2010).
- Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R, Wang H, Mueller C, Sena-Esteves M, Brown Jr RH, Xu Z and Gao G. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther 19: 1440-8 (2011).
- Qiu L, Rivera-Pérez JA and Xu Z. A non-specific effect associated with conditional transgene expression based on Cre-loxP strategy in mice. PLoS One, 6(5):e18778. PMCID: PMC3091857 (2011).
- Yang C, Qiu L, Xu Z (2011) Specific gene silencing using RNAi in cell culture. Methods Mol Biol 793:457-477 (2011).
- Wu CH, Fallini C, Ticozzi N, Keagle PJ, Sapp PC, Piotrowska K, Lowe P, Koppers M, McKenna-Yasek D, Baron DM, Kost JE, Gonzalez-Perez P, Fox AD, Adams J, Taroni F, Tiloca C, Leclerc AL, Chafe SC, Mangroo D, Moore MJ, Zitzewitz JA, Xu ZS, van den Berg LH, Glass JD, Siciliano G, Cirulli ET, Goldstein DB, Salachas F, Meininger V, Rossoll W, Ratti A, Gellera C, Bosco DA, Bassell GJ, Silani V, Drory VE, Brown RH, Jr., Landers JE (2012) Mutations in the profilin 1 gene cause familial amyotrophic lateral sclerosis. Nature 488:499-503 (2012).
- Gibson GE, Chen HL, Xu H, Qiu L, Xu Z, Denton TT, Shi Q. Deficits in the mitochondrial enzyme α-ketoglutarate dehydrogenase lead to Alzheimer's disease-like calcium dysregulation. Neurobiol Aging 33: 1121.e13-24 (2012).
- Xu ZS (2012) Does a loss of TDP-43 function cause neurodegeneration? Mol Neurodegener 7:27. Attained “Highly accessed” designation. (2012).
- Qiu L, Qiao T, Beers M, Tan W, Wang H, Yang B, Xu Z. Widespread aggregation of mutant VAPB associated with ALS does not cause motor neuron degeneration or modulate mutant SOD1 aggregation and toxicity in mice. Mol Neurodegener 8:1. (2013).
- Li S, Sheng J, Hu JK, Yu W, Kishikawa H, Hu MG, Shima K, Wu D, Xu Z, Xin W, Sims KB, Landers JE, Brown RH, Jr., Hu GF (2013) Ribonuclease 4 protects neuron degeneration by promoting angiogenesis, neurogenesis, and neuronal survival under stress. Angiogenesis 16: 387-404 (2013).
- Broering TJ, Wang H, Boatright NK, Wang Y, Baptista K, Shayan G, Garrity KA, Kayatekin C, Bosco DA, Matthews CR, Ambrosino DM, Xu Z, Babcock GJ. Identification of human monoclonal antibodies specific for human SOD1 recognizing distinct epitopes and forms of SOD1. PLoS One 8: e61210 (2013).
- Bi F, Huang C, Tong J, Qiu G, Huang B, Wu Q, Li F, Xu Z, Bowser R, Xia XG, Zhou H. Reactive astrocytes secrete lcn2 to promote neuron death. Proc Natl Acad Sci USA 110:4069-4074. PMID: 23431168 (2013).
- Cheng W, Ip YT and Xu Z. Gudu, an armadillo repeat-containing protein, is required for spermatogenesis in Drosophila. Gene 531: 294-300 (2013).
- Hongyan Wang, Bin Yang,Linghua Qiu, Chunxing Yang, Joshua Kramer, Qin Su, Yansu Guo, Robert H. Brown, Jr., Guangping Gao and Zuoshang Xu. Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Gen 23: 668-681 (2014).
- 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. Partial loss of TDP-43 function causes phenotypes of amyotrophic lateral sclerosis. PNAS 111(12): E1121–E1129 (2014).
- An T, Shi P, Duan W, Zhang S, Yuan P, Li Z, Wu D, Xu Z, Li C, Guo Y. Oxidative stress and autophagic alteration in brainstem of SOD1-G93A mouse model of ALS. Mol Neurobiol 49:1435-1448 (2014).
- Bin Yang, Shaoyong Li, Hongyan Wang, Yansu Guo, Dominic J. Gessler, Chunyan Cao, Qin Su, Joshua Kramer, Li Zhong, Seemin Seher Ahmed, Hongwei Zhang, Ran He, Ronald C. Desrosiers, Robert Brown, Zuoshang Xu* and Guangping Gao*. Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10. Mol Ther 22: 1299–1309. *corresponding authors (2014).
- Xu Z, Yang C. TDP-43—The key to understanding amyotrophic lateral sclerosis. Rare Diseases 2:e944443 (2014).
- Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G, Sena-Esteves M. Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan. Hum Mol Genet 24:4353-4364 (2015).
- Guo Y, Wang D, Qiao T, Yang C, Su Q, Gao G, Xu Z. 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 (2016).
- Yang C, Danielson EW, Qiao T, Metterville J, Brown RH, Jr., Landers JE, Xu Z. Mutant PFN1 causes ALS phenotypes and progressive motor neuron degeneration in mice by a gain of toxicity. Proc Natl Acad Sci U S A (2016).
- Yang K, Gao Y, Yang M, Xu Z, Chen Q. Creating conditional dual fluorescence labeled transgenic animals for studying function of small noncoding RNAs. Connect Tissue Res 58:103-115 (2017).
- Zhao L, Wu X, Wang X, Duan C, Wang H, Punjabi A, Zhao Y, Zhang Y, Xu Z, Gao H and Han G. Development of Excipient-Free Freeze-Dryable Unimolecular Hyperstar Polymers for Efficient siRNA Silencing. Macro Letters 6: 700-704 (2017).
- Li D, Liu C, Yang C, Wang D, Wu D, Qi Y, Su Q, Gao G, Xu Z, Guo Y.Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALS.Neuroscience 365:192-205 (2017)
- Li D, Li Y, Tian Y, Xu Z*, Guo Y*. Direct Intrathecal Injection of Recombinant Adeno-associated Viruses in Adult Mice. J Vis Exp (2019). PMID: 30829325.
- Yang C, Qiao T, Yu J, Wang H, Guo Y, Salameh J, Metterville J, Parsi S, Yusuf I, Brown RH, Cai H, Xu Z. Low-level overexpression of wild type TDP-43 causes late-onset, progressive neurodegeneration and paralysis in mice. PLoS One (2022). PMID: 35113871.
- Lee PTJ, Kennedy Z, Wang Y, Lu Y, Cefaliello C, Uyan O, Song CQ, Godinho BM, Xu Z, Rusckowski M, Xue W, Brown Jr. RH. Imaging net retrograde axonal transport in vivo: a physiological biomarker. Ann Neurol (2022). PMID: 35178738.
- Yusuf IO, Qiao T, Parsi S, Tilvawala R, Thompson PR, Xu Z. Protein citrullination marks myelin protein aggregation and disease progression in mouse ALS models. Acta Neuropathol Commun (2022). PMID: 36076282.
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