Published Research on ALS & Motor Neuron Disorders

2014

Ravits J: Focality, stochasticity and neuroanatomic propagation in ALS pathogenesis. Experimental Neurology, 2014 Aug 6. pii: S0014-4886(14)00249-0. doi: 10.1016/j.expneurol.2014.07.021. [Epub ahead of print] PMID: 25108067
Bruijn L, Cudkowicz M and the ALS Clinical Trials Working Group (including Ravits JM): Opportunities for improving therapy development in ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 2014; 15: 169-173.
Cady, J, Koval, E, Benitez, B, Zaidman, C, Jockel-Balsarotti, J, Allred, P, Baloh, B, Ravits, J, Simpson, E, Appel, S, Pestronk, A, Goate A, Miller, T, Cruchaga,C, Harms, M: The TREM2 variant p.R47H is a risk factor for sporadic amyotrophic lateral sclerosis. JAMA Neurology (2014 Feb 17. doi: 10.1001/jamaneurol.2013.6237.) PMID: 24535663
Bruijn L, Cudkowicz M and the ALS Clinic Trials Working Group (incl Ravits, J): Opportunities for improving therapy development in ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2014, 2013; Early Online: 1-5.
Cortes, C.J., Ling, S.-C., Guo, L, Hung, G., Tsunemi, T., Ly, L., Lopez, E., Sopher, B.L., Bennett, C.F., Shelton, D., Cleveland, D.W. and La Spada, A. (2014). Silencing muscle expression of mutant androgen receptor protein completely rescues systemic and motor neuron disease phenotypes in a Spinal & Bulbar Muscular Atrophy mouse model. Neuron 82, 295-307.
Winkler, E.A., Sengill, J.D., Sagare, A.P., Zhao, Z., Ma, Q., Zuniga, E., Want, Y., Zhong, Z., Sullivan, J.S., Griffin, J.H., Cleveland, D.W. and Zlokovic, B.V. (2014). Blood-spinal cord barrier disruption contributes to early motor neuron degeneration in ALS model mice. Proc. Natl. Acad. Sci. 111, E1035-E1042.
Crotti, A., Benner, C., Kerman, B., Lagier-Tourenne, C., Kordasiewicz, H., Pollard, J.W., Zuccato, C., Cattaneo, E., Gage, F.H., Cleveland, D.W., and Glass, C.K. (2014). Mutant Huntingtin promotes cell-autonomous microglia activation and neuro-inflammation via myeloid lineage-determining factors PU.1-C/EBP. Nat. Neurosci., 17, 513-521.
Lobsiger, C.S. and Cleveland, D.W. (2014). Reply to Woodruff et al.: C1q and C3-dependent complement pathway activation does not contribute to disease in SOD1 mutant ALS mice. Proc. Natl. Acad. Sci. 111, E5.
Meyer, K., Ferraiuolo, L., Miranda, C.J., Likhite, S., McElroy, S., Renusch, S., Ditsworth, D., Lagier-Tournenne, C., Smith, R.A., Ravits, J., Burghes, A., Shaw, P.J., Cleveland, D.W., Kolb, S.J. and Kaspar, B.K (2014). Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS. Proc. Natl. Acad. Sci. 111, 829-832.

2013

Song F, Chiang P, Ravits J, Loeb JA. Activation of microglial neuregulin1 signaling in the corticospinal tracts of ALS patients with upper motor neuron signs. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Nov 14. PMID:24229388.
Sareen D, O'Rourke JG, Meera P, Muhammad AK, Grant S, Simpkinson M, Bell S, Carmona S, Ornelas L, Sahabian A, Gendron T, Petrucelli L, Baughn M, Ravits J, Harms MB, Rigo F, Bennett CF, Otis TS, Svendsen CN, Baloh RH: Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion. Sci Transl Med. 2013 Oct 23;5(208):208ra149. PMID: 24154603.
Ravits J et al: De-cyphering ALS: What phenotype, neuropathology and genetics are telling us about pathobiology. Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14 Suppl 1:5-18. PMID: 23678876.
Harms, MB, Cady, J, Zaidman, C, Cooper, P, Bali, T Allred, P, Cruchaga, C, Baugh M,, Libby RT, Pestronk, A, Goate, A, Ravits, J, Baloh, RH: Lack of C9ORF72 coding mutations supports a gain of function for repeat expansions in ALS. Neurobiol Aging. 2013 Apr 16. doi:pii: S0197-4580(13)00106-1 PMID: 23597494
Benatar M, Wuu J, and Ravits J: Opportunity and Innovation in Studying Pre-Symptomatic ALS. Muscle Nerve. 2013 May;47(5):629-31 PMID: 23494825
Turner MR, Hardiman O, Benatar M, Brooks BR, Chio A, de Carvalho M, Ince PG, Lin C, Miller RG, Mitsumoto H, Nicholson G, Ravits J, Shaw PJ, Swash M, Talbot K, Traynor BJ, Van den Berg LH, Veldink JH, Vucic S, Kiernan MC. Controversies and priorities in amyotrophic lateral sclerosis. Lancet Neurol. 2013 Mar;12(3):310-22.
Lobsiger, C., Boillee, S., Sun, S., Khan, A.M., McAlonis-Downes, M., Lewcock, J. and Cleveland, D.W. (2013). C1q induction and activation of the classic complement pathway do not contribute to ALS toxicity in mutant SOD1 mice. Proc. Natl. Acad. Sci. 110, E4385-E4392.
Lagier-Tourenne, C., Baughn, M., Rigo, F., Sun, Shuying, Liu,P., Li, H.-R., Jiang, Jie, Watt, A., Chun, Seung, Katz, Melanie, Qiu, J., Ling, S.-C., Zhu, Q., Polymenidou, M., Drenner, K., Artates, J.W., McAlonis, M.M., Hung, G., Markmiller, S., Hutt, K.R., Pizzo, D., Baloh, R.H., Vandenberg, S., Yeo, G.W., Fu, X-D., Bennett, C.F., Cleveland, D.W.* and Ravits, J.* (2013). Targeted degradation of sense and antisense C9orf72 nuclear RNA foci as therapy for amyotrophic lateral sclerosis and frontotemporal dementia. Proc. Natl. Acad. Sci 110, E4530-E4539.
Foust, K.D., Salazar, D.L., Likhite, S., Ferraiuolo, L., Ditsworth, D., Ilieva, H., Meyer, K., Schmelzer, L., Braun, L., Cleveland, D.W.* and Kaspar, B.K.* (2013). Therapeutic delivery of AAV9-mediated SOD1 suppression slows disease progression and extends survival in two mouse models of ALS. Molec. Therapy 21, 2148-2159.
Parone, P.A., Da Cruz, S., Han, J.S., McAlonis-Downes, M., Vetto, A.P., Lee, S.K., Tseng, E., and Cleveland, D.W. (2013). Enhancing mitochondrial calcium buffering capacity reduces aggregation of misfolded SOD1 and motor neuron cell death without extending survival in mouse models of inherited amyotrophic lateral sclerosis. J. Neurosci. 33, 4657-4671.
Ling, S.C., Polymenidou, M., and Cleveland, D.W. (2013). Converging mechanisms in ALS and FTD: disrupted RNA and protein homeostasis. Neuron 79, 416-438.
Kang, S.H., Li, Y., Fukaya, M., Lorenzini, I., Cleveland, D.W., Ostrow, L.W., Rothstein, J.D., and Bergles, D.E. (2013).Degeneration and impaired regeneration of gray matter oligodendrocytes in amyotrophic lateral sclerosis. Nat. Neurosci. 16, 571-579.
Arnold, E.S., Ling, S.C., Huelga, S.C., Lagier-Tourenne, C., Polymenidou, M., Ditsworth, D., Kordasiewicz, H.B., McAlonis-Downes, M., Platoshyn, O., Parone, P.A., Da Cruz, S., Clutario, K.M., Swing, D., Tessarollo, L., Marsala, M., Shaw, C.E., Yeo, G.W., and Cleveland, D.W. (2013). ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43. Proc. Natl. Acad. Sci. 110, E736-E745.

2012

Yu, D., Pendergraff, H., Liu, J., Kordasiewicz, H.B., Cleveland, D.W., Swayze, E.E., Lima, W.F., Crooke, S.T., Prakash, T.P., and Corey, D.R. (2012). Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 150, 895-908.
Sun, S. and Cleveland, D.W. (2012). TDP-43 toxicity and the usefulness of junk. Nat. Genet. 44, 1289-1291.
Polymenidou, M., Lagier-Tourenne, C., Hutt, K.R., Bennett, C.F., Cleveland, D.W., and Yeo, G.W. (2012). Misregulated RNA processing in amyotrophic lateral sclerosis. Brain Res. 1462, 3-15.
Polymenidou, M. and Cleveland, D.W. (2012). Prion-like spread of protein aggregates in neurodegeneration. J. Exp. Med. 209, 889-893.
Lagier-Tourenne, C., Polymenidou, M., Hutt, K.R., Vu,A. Q., Baughn, M., Huelga, S.C., Clutario, K.M., Ling, S.C., Liang, T.Y., Mazur, C., Wancewicz, E., Kim, A.S., Watt, A., Freier, S., Hicks, G.G., Donohue, J.P., Shiue, L., Bennett, C.F., Ravits, J., Cleveland, D.W.*, and Yeo, G.W.* (2012). Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs. Nat. Neurosci. 15, 1488-1497.
Kordasiewicz, H.B., Stanek, L.M., Wancewicz, E.V., Mazur, C., McAlonis, M.M., Pytel, K.A., Artates, J.W., Weiss, A., Cheng, S.H., Shihabuddin, L.S., Hung, G., Bennett, C.F., and Cleveland, D.W. (2012).Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 74, 1031-1044.
Hefferan, M.P., Galik, J., Kakinohana, O., Sekerkova, G., Santucci, C., Marsala, S., Navarro, R., Hruska-Plochan, M., Johe, K., Feldman, E., Cleveland, D.W., and Marsala, M. (2012). Human neural stem cell replacement therapy for amyotrophic lateral sclerosis by spinal transplantation. PLoS. One. 7, e42614.
Da Cruz, S., Parone, P.A., Lopes, V.S., Lillo, C., McAlonis-Downes, M., Lee, S.K., Vetto, A.P., Petrosyan, S., Marsala, M., Murphy, A.N., Williams, D.S., Spiegelman, B.M., and Cleveland, D.W. (2012). Elevated PGC-1alpha activity sustains mitochondrial biogenesis and muscle function without extending survival in a mouse model of inherited ALS. Cell Metab 15, 778-786.
Song F, Chiang P, Wang J, Ravits J and Loeb JA: Aberrant neuregulin1 signaling in amyotrophic lateral sclerosis. J Neuropathol Exp Neurol. 2012 Feb;71(2):104-15.

2011

Vande, V.C., McDonald, K.K., Boukhedimi, Y., McAlonis-Downes, M., Lobsiger, C.S., Bel, H.S., Zandona, A., Julien, J.P., Shah, S.B., and Cleveland, D.W. (2011). Misfolded SOD1 associated with motor neuron mitochondria alters mitochondrial shape and distribution prior to clinical onset. PLoS. One. 6, e22031.
Polymenidou, M., Lagier-Tourenne, C., Hutt, K.R., Huelga, S.C., Moran, J., Liang, T.Y., Ling, S.C., Sun, E., Wancewicz, E., Mazur, C., Kordasiewicz, H., Sedaghat, Y., Donohue, J.P., Shiue, L., Bennett, C.F., Yeo, G.W., and Cleveland, D.W. (2011). Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43. Nat. Neurosci. 14, 459-468.
Polymenidou, M. and Cleveland, D.W. (2011). The seeds of neurodegeneration: prion-like spreading in ALS. Cell 147, 498-508.
Da Cruz, S. and Cleveland, D.W. (2011). Understanding the role of TDP-43 and FUS/TLS in ALS and beyond. Curr. Opin. Neurobiol. 21, 904-919.

2010

Ling, S.C., Albuquerque, C.P., Han, J.S., Lagier-Tourenne, C., Tokunaga, S., Zhou, H., and Cleveland, D.W. (2010). ALS-associated mutations in TDP-43 increase its stability and promote TDP-43 complexes with FUS/TLS. Proc. Natl. Acad. Sci. 107, 13318-13323.
Li, Q., Vande, V.C., Israelson, A., Xie, J., Bailey, A.O., Dong, M.Q., Chun, S.J., Roy, T., Winer, L., Yates, J.R., Capaldi, R.A., Cleveland, D.W., and Miller, T.M. (2010). ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import. Proc. Natl. Acad. Sci. 107, 21146-21151.
Lagier-Tourenne, C. and Cleveland, D.W. (2010). Neurodegeneration: An expansion in ALS genetics. Nature 466, 1052-1053.
Lagier-Tourenne, C., Polymenidou, M., and Cleveland, D.W. (2010). TDP-43 and FUS/TLS: emerging roles in RNA processing and neurodegeneration. Hum. Mol. Genet. 19, R46-R64.
Israelson, A., Arbel, N., Da, C.S., Ilieva, H., Yamanaka, K., Shoshan-Barmatz, V., and Cleveland, D.W. (2010). Misfolded mutant SOD1 directly inhibits VDAC1 conductance in a mouse model of inherited ALS. Neuron 67, 575-587.
Lagier-Tourenne C, Polymenidou M, Cleveland DW. TDP-43 and FUS/TLS: emerging roles in RNA processing and neurodegeneration. Hum Mol Genet. 2010 Apr 15;19(R1):R46-64. Epub 2010 Apr 15.
Bodansky A, Kim H, Tempest L, Velagupudi A, Libby RT, Ravits J: TDP-43 and ubiquitinated cytoplasmic aggregates in ALS are low frequency and widely distributed in the lower motor neuron columns independent of disease spread. Amyotroph Lateral Scler. 2010 May 3;11(3):321-7.
Rabin S, Kim H, Baughn M, Libby R, Kim Y, Fan Y, La Spada A, Libby RT, Stone B, Ravits J: Sporadic ALS has compartment-specific aberrant exon splicing and perturbation of cell-matrix adhesion biology. Human Molecular Genetics 2010;19:313-328.

2009

Ravits J, LaSpada A: Motor phenotype heterogeneity, focality, and spread in ALS: deconstructing motor neuron degeneration. Neurology 2009; 73: 805- 811
Kim YJ, Fan Y, Laurie P, 'Hugo' Kim JM, Ravits J: No evidence of HIV pol gene in spinal cord tissues in sporadic ALS by real-time RT-PCR. Amyotroph Lateral Scler. 2009;Mar 25:1-9.
Ilieva H, Polymenidou M, Cleveland DW. Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond. J Cell Biol. 2009 Dec 14;187(6):761-72.
Zhong Z, Ilieva H, Hallagan L, Bell R, Singh I, Paquette N, Thiyagarajan M, Deane R, Fernandez JA, Lane S, Zlokovic AB, Liu T, Griffin JH, Chow N, Castellino FJ, Stojanovic K, Cleveland DW, Zlokovic BV. Activated protein C therapy slows ALS-like disease in mice by transcriptionally inhibiting SOD1 in motor neurons and microglia cells. J Clin Invest. 2009 Nov;119(11):3437-49. doi: 10.1172/JCI38476. Epub 2009 Oct 19.
Lagier-Tourenne C, Cleveland DW. Rethinking ALS: the FUS about TDP-43. Cell. 2009 Mar 20;136(6):1001-4. Review.
Lobsiger CS, Boillee S, McAlonis-Downes M, Khan AM, Feltri ML, Yamanaka K, Cleveland DW. Schwann cells expressing dismutase active mutant SOD1 unexpectedly slow disease progression in ALS mice. Proc Natl Acad Sci U S A. 2009 Mar 17;106(11):4465-70.
Garcia ML, Rao MV, Fujimoto J, Garcia VB, Shah SB, Crum J, Gotow T, Uchiyama Y, Ellisman M, Calcutt NA, Cleveland DW. Phosphorylation of highly conserved neurofilament medium KSP repeats is not required for myelin-dependent radial axonal growth. J Neurosci. 2009 Feb 4;29(5):1277-84.
Cleveland DW, Yamanaka K, Bomont P. Gigaxonin controls vimentin organization through a tubulin chaperone-independent pathway. Hum Mol Genet. 2009 Apr 15;18(8):1384-94.

2008

Xiao S, Tjostheim S, Horne P, Fan Y, Ravits J, Strong M, Fan Y, Robertson J: An aggregate-inducing peripherin isoform generated through intron retention is upregulated in amyotrophic lateral sclerosis and associated with disease pathology. J Neurosci. 2008;28(8):1833-40.
Ilieva HS, Yamanaka K, Malkmus S, Kakinohana O, Yaksh T, Marsala M, Cleveland DW. Mutant dynein (Loa) triggers proprioceptive axon loss that extends survival only in the SOD1 ALS model with highest motor neuron death. Proc Natl Acad Sci U S A. 2008 Aug 26;105(34):12599-604.
Polymenidou M, Cleveland DW. Motor neuron disease: The curious ways of ALS. Nature. 2008 Jul 17;454(7202):284-5.
Yamanaka K, Boillee S, Roberts EA, Garcia ML, McAlonis-Downes M, Mikse OR, Cleveland DW, Goldstein LS. Mutant SOD1 in cell types other than motor neurons and oligodendrocytes accelerates onset of disease in ALS mice. Proc Natl Acad Sci U S A. 2008 May 27;105(21):7594-9.
Vande Velde C, Miller TM, Cashman NR, Cleveland DW. Selective association of misfolded ALS-linked mutant SOD1 with the cytoplasmic face of mitochondria. Proc Natl Acad Sci U S A. 2008 Mar 11;105(10):4022-7. Yamanaka K, Chun SJ, Boillee S, Fujimori-Tonou N, Yamashita H, Gutmann DH, Takahashi R, Misawa H, Cleveland DW. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat Neurosci. 2008 Mar;11(3):251-3.
Boillée S, Cleveland DW. Revisiting oxidative damage in ALS: microglia, Nox, and mutant SOD1. J Clin Invest. 2008 Feb;118(2):474-8.

2007

Gordon PH, Moore DH, Miller RG et al (incl Ravits J): Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomized trial. Lancet Neurology 2007;6:1045-53.
Ravits J, Laurie P, Fan Y, and Moore DH: Implications of ALS focality: rostral-caudal distribution of lower motor neuron loss postmortem. Neurology 2007;68:1576-1582.
Ravits J, Paul P, Jorg C: Focality of upper and lower motor neuron degeneration at the clinical onset of ALS. Neurology 2007;68:1571-1575.
Mackenzie IRA, Bigio EH, Ince PG, Geser F, Neumann M, Cairns NJ, Kwong LW, Forman MS, Ravits J, Shaw PJ, Stewart H, McClusky L, Kretzschmar HA, Monoranu C, Highley R, Kirby J, Siddique T, Lee V M-Y, Trojanowski JQ: Pathological TDP-43 distinguishes sporadic ALS from ALS with SOD-1 mutations. Ann Neurol 2007;61:427-434.
Lobsiger CS, Cleveland DW. Glial cells as intrinsic components of non-cell-autonomous neurodegenerative disease. Nat Neurosci. 2007 Nov;10(11):1355-60. Review.
Lobsiger CS, Boillée S, Cleveland DW. Toxicity from different SOD1 mutants dysregulates the complement system and the neuronal regenerative response in ALS motor neurons. Proc Natl Acad Sci U S A. 2007 May 1;104(18):7319-26.

2006

Boillée S, Vande Velde C, Cleveland DW. ALS: a disease of motor neurons and their nonneuronal neighbors. Neuron. 2006 Oct 5;52(1):39-59. Review.
Boillée S, Yamanaka K, Lobsiger CS, Copeland NG, Jenkins NA, Kassiotis G, Kollias G, Cleveland DW. Onset and progression in inherited ALS determined by motor neurons and microglia. Science. 2006 Jun 2;312(5778):1389-92.
Garcia, M.L., Singleton, A., Hernandez, D., Ward, C.M., McClure, J., Evey, C., Sapp, P.A., Hardy, J.A., Brown, R.H., Jr., and Cleveland, D.W. (2006). Mutations in neurofilament genes are not a primary cause of non-SOD1 mediated Amyotrophic Lateral Sclerosis. Neurobiol. Dis., 21, 102-109.

2005

Ravits J: Sporadic amyotrophic lateral sclerosis-a hypothesis of persisting enteroviral infection. Amyotrophic Lateral Sclerosis and other motor neuron disorders 2005;6:77-87.
Andersen PM, Sims KB, Xin WW, Kiely R, O'Neill G, Ravits J, Pioro E, Harati Y, Brower RD, Levine JS, Heinicke HU, Seltzer W, Boss M, Brown RH. Sixteen novel mutations in the gene encoding CuZn-superoxide dismutase in ALS. Amyotrophic Lateral Sclerosis and other motor neuron disorders, 2003; 2: 62-73.
Weiss MD, Ravits JM, Schuman N, Carter GT: A4V superoxide dismutase mutation in apparently sporadic ALS. Amyotrophic Lateral Sclerosis and other motor neuron disorders 2006 Mar;7(1):61-3.
Miller, T.M. and Cleveland, D.W. (2005). Treating neurodegenerative disease with antibiotics. Science 307, 361-362.
Miller, T.M., Kaspar, B.K., Kops, G.J., Yamanka, K., Christian, L.J., Gage, F.H. and Cleveland, D.W. (2005). Virus-delivered Small RNA Silencing Strength in Amyotrophic Lateral Sclerosis. Ann. Neurol. 57, 773-776. Vande Velde, C. and Cleveland, D.W. (2005). VEGF - multitasking in ALS. Nat. Neurosci., 8, 5-7.

2004

Boillee, S. and Cleveland, D.W. (2004). Gene therapy for ALS delivers. Trends Neurosci.. 27, 235-238.
Bruijn, L.I., Miller, T.I. and Cleveland, D.W. (2004). Unraveling the mechanisms involved in motor neuron degeneration in ALS. Ann. Rev. Neurosci. 27, 723-749.
Liu, J., Lillo, C., Jonsson, P.A., C. Vande Velde, Miller, T.M., Subramaniam, J.R., Rothstein, J.D., Marklund, S., Andersen, P.M., Brannstrom, T., Wong, P.C., Williams, D.S. and Cleveland, D.W. (2004). Toxicity of familial ALS-linked SOD1 mutants from selective recruitment to spinal mitochondria. Neuron 43, 5-17.
Vande Velde, C., Garcia, M.L., Yin, X., Trapp, B.D., and Cleveland, D.W. (2004). The neuroprotective factor Wlds does not attenuate mutant SOD1-mediated motor neuron disease. NeuroMol. Med. 5, 193-204.

2003

Bryan WW, Hoagland RJ, and The Syntex/Synergen Neuroscience Joint Venture rhCNTF ALS Study Group (including Ravits J): Can we eliminate placebo in ALS clinical trials? ALS and other Motor Neuron Diseases 2003;4:11-15.
Clement, A., Nguyen, M, Roberts, E.A., Garcia, M.L., Boillee, S., Rule, M., McMahon, A.P. Doucette, W., Siwek, D., Ferrante, R.J., Brown, R.H., Julien, J-P., Goldstein, L.S.B. and Cleveland, D.W. (2003). Wild type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice. Science 302, 113-117.
Garcia, M.L., Lobsiger, C.S., Shah, S.B., Deerinck, T.J., Crum, J., Yound, D., Ward, C.M., Crawford, T.O., Gotow, T., Ellisman, M.H., Calcutt, N.A. and Cleveland, D.W. (2003). NF-M is an essential target for the mylein-directed "outside-in" signaling cascade that mediates radial axonal growth. J. Cell Biol. 163, 1011-1020.
Miller, T.M. and Cleveland, D.W. (2003). Has gene therapy arrived for ALS? Nat. Med. 9, 1256-1257.
Yamanaka, K., VandeVelde, C., Bertini, E., Boespflug-Tanguy, O. and Cleveland, D.W. (2003). Unstable mutants in the peripheral endosomal membrane component ALS2 cause early onset motor neuron disease. Proc. Natl. Acad. Sci. 100, 16041-16046.

2002

Howland, D.S., Liu, J., She, Y., Goad, B., Margakis, N.J., Kim, B., Erickson, J., Kulik, J., DeVito, L., Psaltis, G., DeGennaro, L.J., Cleveland, D.W. and Rothstein, J.D. (2002). Focal loss of the glutatmate transporter EAAT2 in a transgenic rat model of SOD1 mutant-mediate Amyotrophic Lateral Sclerosis (ALS). Proc. Natl. Acad. Sci. U.S.A. 99, 1604-1609.
Raoul, C., Estevez, A.G., Nishimune, H., Cleveland, D.W., deLapeyriere, Henderson, C.E., O., Haase, G and Pettmann, B. (2002). Motoneuron death triggered by a specific pathway downstream of Fas: potentiation by ALS-linked SOD1 mutations. Neuron 35, 1067-1083.
Subramaniam, J.R., Lyons, W.E., Liu, J., Bartnikas, T.B., Rothstein, J.D., Price, D.L., Cleveland, D.W., Gitlin, J.D., and Wong, P.C. (2002) Mutant SOD1 causes motor neuron disease independent of copper chaperone-mediated copper loading. Nat. Neurosci. 5, 301-307.

2001

Miller RG, Moore D, Gelinas DF et al (including Ravits J): Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 2001;56:843-848.
Cleveland, D.W. and Rothstein, J.D. (2001) From Charcot to Lou Gehrig: deciphering selective motor neuron death in ALS. Nat. Neurosci. Rev. 2, 806-819.
Kato, S., Nakashima, K., Horiuchi, S., Nagai, R., Cleveland, D.W., Liu, J., Hirano,A., Takikawa, M., Kato, M., Nakano, I., Sakoda, S., Asayama, K., and Ohama, E. (2001). Formation of advanced glycation end-product modified superoxide dismutase-1 (SOD1) is one of the mechanisms responsible for inclusions common to familial amyotrophic lateral scleorsis patients with SOD1 gene mutation and transgenic mice expressing human SOD1 gene mutation. Neuropathology 21, 67-81.
Skene, J.H.P. and Cleveland, D.W. (2001). Hypoxia and Lou Gehrig. Nature Genetics 28, 107-108.

2000

Fernandez M, McClain ME, Martinez RA, Snow K, Lipe H, Ravits J, Bird TD, & LaSpada AR: Late-onset SCA2: 33 CAG repeats are sufficient to cause disease. Neurology 2000;55(4):569-572.
Cleveland, D.W. and Liu, J. (2000). Oxidation versus aggregation -- how do SOD1 mutants cause ALS? Nature Medicine 12, 1320-1321.
Kato, S., Horiuchi, S., Liu, J., Cleveland, D.W., Shibata, N., Nakashima, K., Nagai, R., Hirano, A., Takikawa, M., Kato, M., Nakano, I. and Ohama, E. (2000) Advanced glycation endproduct-modified superoxide dismutase 1 (SOD1)-positive inclusion are common to familial amyotrophic lateral sclerosis patients with SOD1 gene mutations and transgenic mice expressing human SOD1 with G85R mutation. Acta Neuropath.100, 490-505.
Pasinelli, P., Houseweart, M.K., Brown, R.H. and Cleveland, D.W. (2000). Caspase-1 and -3 are sequentially activated in motor neuron death in Cu,Zn superoxide dismutasemediated familial amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci. 97, 13901-13906.
Williamson, T.L., Corson, L.B., Huang, L., Burlingame, A., Liu, J., Bruijn, L.I. and Cleveland, D.W. (2000). Toxicity of ALS-Linked SOD1 Mutants. Science 288, 399-400.

1999

Cleveland, D.W. (1999). From Charcot to SOD1: mechanisms of selective motor neuron death in ALS. Neuron 24, 515-520.
Houseweart, M.K. and Cleveland, D.W. (1999). Bcl-2 Overexpression Does Not Protect Neurons from Mutant Neurofilament-Mediated Motor Neuron Degeneration. J. Neurosci. 9, 6446-6456.
Ratovitski, T., Corson, L.B., Strain, J., Wong, P.C., Cleveland, D.W., Culotta, V.C. and Borchelt, D.R. (1999). Variation in the biochemical/biophysical properties of mutant superoxide dismutase 1 enzymes and the rate of disease progression in familial amyotrophic lateral sclerosis kindreds. Hum. Mol. Genet. 8, 1451-1460.
Rothstein, J.D., Dykes-Hoberg, M., Corson, L.B., Becker, M., Cleveland, D.W., Price, D.L., Culotta, V.C., and Wong, P.C. (1999). The copper chaperone CCS is abundant in neurons and astrocytes in human and rodent brain. J. Neurochem. 72, 422-429.
Williamson, T.L. and Cleveland, D.W. (1999). Slowing of axonal transport is a very early event in the toxicity of ALS-linked SOD1 mutants to motor neurons. Nature Neurosci. 2, 50-56.

1998

Ravits J: Neurology epitome: update on treatment of peripheral neuropathy. West J Med 168:268-9, 1998.
Ross MA, Miller RG Berchert L et al and the rhCNTF ALS Study Group (including Ravits J): Toward earlier diagnosis of amyotrophic lateral sclerosis: Revised criteria. Neurology, 1998;50:768-772.
Borchelt, D.R., Wong, P.C., Becher, M.W., Parto, C.A., Lee, M.K. Xu, Z.-S., Tinakaran, G., Jenkins, N.A., Copeland, N.G., Sisodia, S.S., Cleveland, D.W., Price, D.L. and Hoffman, P.N. (1998). Axonal transport of mutant superoxide dismutase 1 and focal axonal abnormalities in the proximal axons of transgenic mice. Neuro. Dis. 5, 27-35.
Bruijn, L.I., Houseweart, M.K., Kato, S., Anderson, K.L., Anderson, S.D., Ohama, E., Reaume, A.G., Scott, R. and Cleveland, D.W. (1998). Aggregation and Motor Neuron Toxicity of an ALS-Linked SOD1 Mutant Independent from Wild-Type SOD1. Science 281, 1851-1854.
Corson, L.B., Strain, J., Culotta, V.C. and Cleveland, D.W. (1998). Chaperone-facilitated copper binding is a property common to several classes of familial amyotrophic lateral sclerosis-linked superoxide dismutase mutants. Proc. Natl. Acad. Sci. 95, 6361-6366.
Eyer, J., Cleveland, D.W., Wong, P.C. and Peterson, A.C. (1998). Pathogenesis of two axonpathies does not require axonal neurofilaments. Nature 391, 584-587.
Fuchs, E. and Cleveland, D.W. (1998). A structural scaffold of intermediate filaments in health and disease. Science 279, 514-519.
Williamson, T.L., Bruijn, L.I., Zhu, Q. Anderson, K.L., Anderson, S.D., Julien, J.-P. and Cleveland, D.W. (1998). Absence of neurofilaments reduces the selective vulnerability of motor neurons and slows disease caused by a familial ALS-linked SOD1 mutant. Proc. Natl. Acad. Sci. 95, 9631-9636.

1997

Hoagland RJ et al and The Syntex/Synergen Neuroscience Joint Venture rhCNTF ALS Study Group (including Ravits J): Reliability of maximal voluntary isometric contraction testing in a multicenter study of patients with amyotrophic lateral sclerosis. Muscle Nerve 1997;20 (6):691-5.
McGuire D et al and the Syntex-Synergen ALS/CNTF Study Group (including Ravits J): A brief quality-of-life measure for ALS clinical trials based on subset of items from the sickness impact profile. J Neurol Sci. 1997;152 Suppl 1:S18-22.
Bruijn, L.I., Becher, M.W., Lee, M.K., Jenkins, N.A., Copeland, N.G., Sisodia, S.S., Rothstein, J.D., Borchelt, D.R., Price, D.L. and Cleveland, D.W. (1997). ALS-Linked SOD1 Mutant G85R Mediates Damage to Astrocytes and Promotes Rapidly Progressive Disease with SOD1-Containing Inclusions. Neuron 18, 327-338.
Bruijn, L.I., Beal, M.F., Becher, M.W., Schultz, J.B., Wong, P.C., Price., D.L. and Cleveland, D.W. (1997). Elevated free nitrotyrosine levels, but not protein-bound nitrotyrosine of hydroxl radicals, throughout amyotrophic lateral sclerosis (ALS)-like disease implicate tyrosine nitration as an aberrant in vivo property of one familial ALS-linked superoxide dismutase 1 mutant. Proc. Natl. Acad. Sci. 94, 7606-7611.