Publications


Neuroinflammatory mechanisms in amyotrophic lateral sclerosis pathogenesis
Thonhoff, JR, Simpson, EP & Appel, SH 2018, Current Opinion in Neurology. DOI: 10.1097/WCO.0000000000000599

Expanded autologous regulatory T-lymphocyte infusions in ALS
Thonhoff, JR, Beers, DR, Zhao, W, Pleitez, M, Simpson, EP, Berry, JD, Cudkowicz, ME & Appel, SH 2018, Neurology - Neuroimmunology Neuroinflammation, vol 5, no. 4.

The role of regulatory T lymphocytes in amyotrophic lateral sclerosis
Beers, DR, Zhao, W & Appel, SH 2018, JAMA Neurology, vol 75, no. 6, pp. 656-658. DOI: 10.1001/jamaneurol.2018.0043

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene
Nicolas, A, Kenna, KP, Renton, AE, Ticozzi, N, Faghri, F, Chia, R, Dominov, JA, Kenna, BJ, Nalls, MA, Keagle, P, Rivera, AM, van Rheenen, W, Murphy, NA, van Vugt, JJFA, Geiger, JT, Van der Spek, RA, Pliner, HA, Shankaracharya, Smith, BN, Marangi, G, Topp, SD, Abramzon, Y, Gkazi, AS, Eicher, JD, Kenna, A, Logullo, FO, Simone, IL, Logroscino, G, Salvi, F, Bartolomei, I, Borghero, G, Murru, MR, Costantino, E, Pani, C, Puddu, R, Caredda, C, Piras, V, Tranquilli, S, Cuccu, S, Corongiu, D, Melis, M, Milia, A, Marrosu, F, Marrosu, MG, Floris, G, Cannas, A, Capasso, M, Caponnetto, C, Mancardi, G, Origone, P, Mandich, P, Conforti, FL, Cavallaro, S, Mora, G, Marinou, K, Sideri, R, Penco, S, Mosca, L, Lunetta, C, Pinter, GL, Corbo, M, Riva, N, Carrera, P, Volanti, P, Mandrioli, J, Fini, N, Fasano, A, Tremolizzo, L, Arosio, A, Ferrarese, C, Trojsi, F, Tedeschi, G, Monsurrò, MR, Piccirillo, G, Femiano, C, Ticca, A, Ortu, E, La Bella, V, Spataro, R, Colletti, T, Sabatelli, M, Zollino, M, Conte, A, Luigetti, M, Lattante, S, Santarelli, M, Petrucci, A, Pugliatti, M, Pirisi, A, Parish, LD, Occhineri, P, Giannini, F, Battistini, S, Ricci, C, Benigni, M, Cau, TB, Loi, D, Calvo, A, Moglia, C, Brunetti, M, Barberis, M, Restagno, G, Casale, F, Marrali, G, Fuda, G, Ossola, I, Cammarosano, S, Canosa, A, Ilardi, A, Manera, U, Grassano, M, Tanel, R, Pisano, F, Mazzini, L, Messina, S, DAlfonso, S, Corrado, L, Ferrucci, L, Harms, MB, Goldstein, DB, Shneider, NA, Goutman, S, Simmons, Z, Miller, TM, Chandran, S, Pal, S, Manousakis, G, Appel, SH, Simpson, E, Wang, L, Baloh, RH, Gibson, S, Bedlack, R, Lacomis, D, Sareen, D, Sherman, A, Bruijn, L, Penny, M, Moreno, CDAM, Kamalakaran, S, Allen, AS, Boone, BE, Brown, R, Carulli, JP, Chesi, A, Chung, WK, Cirulli, ET, Cooper, GM, Couthouis, J, Day-Williams, AG, Dion, PA, Gitler, AD, Glass, JD, Han, Y, Harris, T, Hayes, SD, Jones, AL, Keebler, J, Krueger, BJ, Lasseigne, BN, Levy, SE, Lu, YF, Maniatis, T, McKenna-Yasek, D, Myers, RM, Petrovski, S, Pulst, SM, Raphael, AR, Ravits, JM, Ren, Z, Rouleau, GA, Sapp, PC, Sims, KB, Staropoli, JF, Waite, LL, Wang, Q, Wimbish, JR, Xin, WW, Phatnani, H, Kwan, J, Broach, JR, Arcila-Londono, X, Lee, EB, Van Deerlin, VM, Fraenkel, E, Ostrow, LW, Baas, F, Zaitlen, N, Berry, JD, Malaspina, A, Fratta, P, Cox, GA, Thompson, LM, Finkbeiner, S, Dardiotis, E, Hornstein, E, MacGowan, DJ, Heiman-Patterson, T, Hammell, MG, Patsopoulos, NA, Dubnau, J, Nath, A, Musunuri, RL, Evani, US, Abhyankar, A, Zody, MC, Kaye, J, Wyman, S, LeNail, A, Lima, L, Rothstein, JD, Svendsen, CN, Van Eyk, J, Maragakis, NJ, Kolb, SJ, Cudkowicz, M, Baxi, E, Wyman, SK, Van Eyk, JE, Benatar, M, Taylor, JP, Wu, G, Rampersaud, E, Wuu, J, Rademakers, R, Züchner, S, Schule, R, McCauley, J, Hussain, S, Cooley, A, Wallace, M, Clayman, C, Barohn, R, Statland, J, Ravits, J, Swenson, A, Jackson, C, Trivedi, J, Khan, S, Katz, J, Jenkins, L, Burns, T, Gwathmey, K, Caress, J, McMillan, C, Elman, L, Pioro, E, Heckmann, J, So, Y, Walk, D, Maiser, S, Zhang, J, Silani, V, Gellera, C, Ratti, A, Taroni, F, Lauria, G, Verde, F, Fogh, I, Tiloca, C, Comi, GP, Sorarù, G, Cereda, C, De Marchi, F, Corti, S, Ceroni, M, Siciliano, G, Filosto, M, Inghilleri, M, Peverelli, S, Colombrita, C, Poletti, B, Maderna, L, Del Bo, R, Gagliardi, S, Querin, G, Bertolin, C, Pensato, V, Castellotti, B, Camu, W, Mouzat, K, Lumbroso, S, Corcia, P, Meininger, V, Besson, G, Lagrange, E, Clavelou, P, Guy, N, Couratier, P, Vourch, P, Danel, V, Bernard, E, Lemasson, G, Laaksovirta, H, Myllykangas, L, Jansson, L, Valori, M, Ealing, J, Hamdalla, H, Rollinson, S, Pickering-Brown, S, Orrell, RW, Sidle, KC, Hardy, J, Singleton, AB, Johnson, JO, Arepalli, S, Polak, M, Asress, S, Al-Sarraj, S, King, A, Troakes, C, Vance, C, de Belleroche, J, ten Asbroek, ALMA, Muñoz-Blanco, JL, Hernandez, DG, Ding, J, Gibbs, JR, Scholz, SW, Floeter, MK, Campbell, RH, Landi, F, Bowser, R, MacGowan, DJL, Kirby, J, Pioro, EP, Pamphlett, R, Broach, J, Gerhard, G, Dunckley, TL, Brady, CB, Kowall, NW, Troncoso, JC, Le Ber, I, Heiman-Patterson, TD, Kamel, F, Van Den Bosch, L, Strom, TM, Meitinger, T, Shatunov, A, Van Eijk, KR, de Carvalho, M, Kooyman, M, Middelkoop, B, Moisse, M, McLaughlin, RL, Van Es, MA, Weber, M, Boylan, KB, Van Blitterswijk, M, Morrison, KE, Basak, AN, Mora, JS, Drory, VE, Shaw, PJ, Turner, MR, Talbot, K, Hardiman, O, Williams, KL, Fifita, JA, Nicholson, GA, Blair, IP, Esteban-Pérez, J, García-Redondo, A, Al-Chalabi, A, Al Kheifat, A, Andersen, P, Chio, A, Cooper-Knock, J, Dekker, A, Drory, V, Redondo, AG, Gotkine, M, Hide, W, Iacoangeli, A, Glass, J, Kenna, K, Kiernan, M, Landers, J, McLaughlin, R, Mill, J, Neto, MM, Pardina, JM, Morrison, K, Newhouse, S, Pinto, S, Pulit, S, Robberecht, W, Shaw, P, Shaw, C, Sproviero, W, Tazelaar, G, Van Damme, P, van den Berg, L, van Eijk, K, van Es, M, van Vugt, J, Veldink, J, Zatz, M, Bauer, DC, Twine, NA, Rogaeva, E, Zinman, L, Brice, A, Goutman, SA, Feldman, EL, Gibson, SB, Ludolph, AC, Andersen, PM, Weishaupt, JH, Trojanowski, JQ, Brown, RH, van den Berg, LH, Veldink, JH, Stone, DJ, Tienari, P, Chiò, A, Shaw, CE, Traynor, BJ, Landers, JE, ITALSGEN Consortium, Genomic Translation for ALS Care (GTAC) Consortium, ALS Sequencing Consortium, NYGC ALS Consortium, Answer ALS Foundation, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, SLAGEN Consortium, French ALS Consortium & Project MinE ALS Sequencing Consortium 2018, Neuron, vol 97, no. 6, pp. 1268-1283.e6. DOI: 10.1016/j.neuron.2018.02.027

An open label study of a novel immunosuppression intervention for the treatment of amyotrophic lateral sclerosis
Fournier, CN, Schoenfeld, D, Berry, JD, Cudkowicz, ME, Chan, J, Quinn, C, Brown, RH, Salameh, JS, Tansey, MG, Beers, DR, Appel, SH & Glass, JD 2018, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, pp. 1-8. DOI: 10.1080/21678421.2017.1421666

Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability
Berry, JD, Paganoni, S, Atassi, N, Macklin, EA, Goyal, N, Rivner, M, Simpson, E, Appel, S, Grasso, DL, Mejia, NI, Mateen, F, Gill, A, Vieira, F, Tassinari, V & Perrin, S 2017, Muscle and Nerve, vol 56, no. 6, pp. 1077-1084. DOI: 10.1002/mus.25733

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
Howard, JF, Utsugisawa, K, Benatar, M, Murai, H, Barohn, RJ, Illa, I, Jacob, S, Vissing, J, Burns, TM, Kissel, JT, Muppidi, S, Nowak, RJ, OBrien, F, Wang, JJ, Mantegazza, R, Mazia, CG, Wilken, M, Ortea, C, Saba, J, Rugiero, M, Bettini, M, Vidal, G, Garcia, AD, Lamont, P, Leong, WK, Boterhoven, H, Fyfe, B, Roberts, L, Jasinarachchi, M, Willlems, N, Wanschitz, J, Löscher, W, De Bleecker, J, Van den Abeele, G, de Koning, K, De Mey, K, Mercelis, R, Wagemaekers, L, Mahieu, D, Van Damme, P, Smetcoren, C, Stevens, O, Verjans, S, DHondt, A, Tilkin, P, Alves de Siqueira Carvalho, A, Hasan, R, Dias Brockhausen, I, Feder, D, Ambrosio, D, Melo, AP, Rocha, R, Rosa, B, Veiga, T, Augusto da Silva, L, Gonçalves Geraldo, J, da Penha Morita Ananias, M, Nogueira Coelho, E, Paiva, G, Pozo, M, Prando, N, Dada Martineli Torres, D, Fernanda Butinhao, C, Coelho, E, Renata Cubas Volpe, L, Duran, G, Gomes da Silva, TC, Otavio Maia Gonçalves, L, Pazetto, LE, Souza Duca, L, Suriane Fialho, TA, Gheller Friedrich, MA, Guerreiro, A, Mohr, H, Pereira Martins, M, da Cruz Pacheco, D, Macagnan, AP, de Cassia Santos, A, Bulle Oliveira, AS, Amaral de Andrade, AC, Annes, M, Cavalcante Lino, V, Pinto, W, Miranda, C, Carrara, F, Souza, I, Genge, A, Massie, R, Campbell, N, Bril, V, Katzberg, H, Soltani, M, Ng, E, Siddiqi, Z, Phan, C, Blackmore, D, Vohanka, S, Bednarik, J, Chmelikova, M, Cierny, M, Toncrova, S, Junkerova, J, Kurkova, B, Reguliova, K, Zapletalova, O, Pitha, J, Novakova, I, Tyblova, M, Wolfova, M, Jurajdova, I, Andersen, H, Harbo, T, Vinge, L, Mogensen, A, Højgaard, J, Witting, N, Autzen, AM, Pedersen, J, Färkkilä, M, Atula, S, Nyrhinen, A, Erälinna, JP, Laaksonen, M, Oksaranta, O, Eriksson, J, Harrison, T, Desnuelle, C, Sacconi, S, Soriani, MH, Decressac, S, Moutarde, J, Lahaut, P, Solé, G, Le Masson, G, Wielanek-Bachelet, AC, Gaboreau, M, Moreau, C, Wilson, A, Vial, C, Bouhour, F, Gervais-Bernard, H, Merle, H, Hourquin, C, Lacour, A, Outteryck, O, Vermersch, P, Zephir, H, Millois, E, Deneve, M, Deruelle, F, Schoser, B, Wenninger, S, Stangel, M, Alvermann, S, Gingele, S, Skripuletz, T, Suehs, KW, Trebst, C, Fricke, K, Papagiannopoulos, S, Bostantzopoulou, S, Vlaikidis, N, Zampaki, M, Papadopoulou, N, Mitsikostas, DD, Kasioti, E, Mitropoulou, E, Charalambous, D, Rozsa, C, Horvath, M, Lovas, G, Matolcsi, J, Szabo, G, Szabadosne, B, Vecsei, L, Dezsi, L, Varga, E, Konyane, M, Gross, B, Azrilin, O, Greenbereg, N, Bali Kuperman, H, Antonini, G, Garibaldi, M, Morino, S, Troili, F, Di Pasquale, A, Filla, A, Costabile, T, Marano, E, Sacca, F, Marsil, A, Puorro, G, Maestri Tassoni, M, De Rosa, A, Bonanno, S, Antozzi, C, Maggi, L, Campanella, A, Angelini, C, Cudia, P, Pegoraro, V, Pinzan, E, Bevilacqua, F, Orrico, D, Bonifati, DM, Evoli, A, Alboini, PE, DAmato, V, Iorio, R, Inghilleri, M, Fionda, L, Frasca, V, Giacomelli, E, Gori, M, Lopergolo, D, Onesti, E, Gabriele, M, Patti, F, Salvatore Caramma, A, Messina, S, Reggio, E, Caserta, C, Uzawa, A, Kanai, T, Mori, M, Kaneko, Y, Kanzaki, A, Kobayashi, E, Masaki, K, Matsuse, D, Matsushita, T, Uehara, T, Shimpo, M, Jingu, M, Kikutake, K, Nakamura, Y, Sano, Y, Nagane, Y, Kamegamori, I, Fujii, Y, Futono, K, Tsuda, T, Saito, Y, Suzuki, H, Morikawa, M, Samukawa, M, Kamakura, S, Shiraishi, H, Mitazaki, T, Motomura, M, Mukaino, A, Yoshimura, S, Asada, S, Kobashikawa, T, Koga, M, Maeda, Y, Takada, K, Takada, MT, Yamashita, Y, Yoshida, S, Suzuki, Y, Akiyama, T, Narikawa, K, Tsukita, K, Meguro, F, Fukuda, Y, Sato, M, Matsuo, H, Fukudome, T, Gondo, Y, Maeda, Y, Nagaishi, A, Nakane, S, Okubo, Y, Okumura, M, Funaka, S, Kawamura, T, Makamori, M, Takahashi, M, Hasuike, T, Higuchi, E, Kobayashi, H, Osakada, K, Taichi, N, Tsuda, E, Hayashi, T, Hisahara, S, Imai, T, Kawamata, J, Murahara, T, Saitoh, M, Shimohama, S, Suzuki, S, Yamamoto, D, Konno, S, Imamura, T, Inoue, M, Murata, M, Nakazora, H, Nakayama, R, Ikeda, Y, Ogawa, M, Shirane, M, Kanda, T, Kawai, M, Koga, M, Ogasawara, J, Omoto, M, Sano, Y, Arima, H, Fukui, S, Shimose, S, Shinozaki, H, Watanabe, M, Yoshikawa, C, van der Kooi, A, de Visser, M, Gibson, T, Maessen, J, de Baets, M, Faber, C, Keijzers, MJ, Miesen, M, Kostera-Pruszczyk, A, Kaminska, A, Kim, BJ, Lee, CN, Koo, YS, Seok, HY, Kang, HN, Ra, HJ, Kim, BJ, Cho, EB, Lee, HL, Min, JH, Seok, J, Koh, DY, Kwon, JY, Lee, JE, Park, SA, Hong, YH, Lim, JS, Kim, MR, Kim, SM, Kim, YH, Lee, HS, Shin, HY, Hwang, EB, Shin, MJ, Sazonov, D, Yarmoschuk, A, Babenko, L, Malkova, N, Melnikova, A, Korobko, D, Kosykh, E, Pokhabov, D, Nesterova, Y, Abramov, V, Balyazin, V, Casasnovas Pons, C, Alberti Aguilo, M, Homedes-Pedret, C, Palacios, NJ, Lazaro, A, Diez Tejedor, E, Fernandez-Fournier, M, Lopez Ruiz, P, Rodriguez de Rivera, FJ, Salvado Figueras, M, Gamez, J, Salvado, M, Cortes Vicente, E, Diaz-Manera, J, Querol Gutierrez, L, Rojas Garcia, R, Vidal, N, Arribas-Ibar, E, Piehl, F, Hietala, A, Bjarbo, L, Lindberg, C, Jons, D, Andersson, B, Sengun, I, Ozcelik, P, Tuga, C, Ugur, M, Boz, C, Altiparmak, D, Gazioglu, S, Ozen Aydin, C, Erdem-Ozdamar, S, Bekircan-Kurt, CE, Yilmaz, E, Acar, NP, Caliskan, Y, Efendi, H, Aydinlik, S, Cavus, H, Semiz, C, Tun, O, Terzi, M, Dogan, B, Onar, MK, Sen, S, Cavdar, TK, Norwood, F, Dimitriou, A, Gollogly, J, Mahdi-Rogers, M, Seddigh, A, Maier, G, Sohail, F, Sathasivam, S, Arndt, H, Davies, D, Watling, D, Rivner, M, Hartmann, JE, Quarles, B, Smalley, N, Amato, A, Cochrane, T, Salajegheh, M, Roe, K, Amato, K, Toska, S, Wolfe, G, Silvestri, N, Patrick, K, Zakalik, K, Katz, J, Miller, R, Engel, M, Bravver, E, Brooks, B, Plevka, S, Burdette, M, Sanjak, M, Kramer, M, Nemeth, J, Schommer, C, Juel, V, Guptill, J, Hobson-Webb, L, Beck, K, Carnes, D, Loor, J, Anderson, A, Lange, D, Agopian, E, Goldstein, J, Manning, E, Kaplan, L, Holzberg, S, Kassebaum, N, Pascuzzi, R, Bodkin, C, Kincaid, J, Snook, R, Guinrich, S, Micheels, A, Chaudhry, V, Corse, A, Mosmiller, B, Ho, D, Srinivasan, J, Vytopil, M, Ventura, N, Scala, S, Carter, C, Donahue, C, Herbert, C, Weiner, E, McKinnon, J, Haar, L, McKinnon, N, Alcon, K, Daniels, K, Sattar, N, Jeffery, D, McKenna, K, Guidon, A, David, W, Dheel, C, Levine-Weinberg, M, Nigro, C, Simpson, E, Appel, SH, Lai, E, Lay, L, Pleitez, M, Halton, S, Faigle, C, Thompson, L, Sivak, M, Shin, S, Bratton, J, Jacobs, D, Brown, G, Bandukwala, I, Brown, M, Kane, J, Blount, I, Freimer, M, Hoyle, JC, Agriesti, J, Khoury, J, Marburger, T, Kaur, H, Dimitrova, D, Mellion, M, Sachs, G, Crabtree, B, Keo, R, Perez, EK, Taber, S, Gilchrist, J, Andoin, A, Darnell, T, Goyal, N, Sakamuri, S, So, YT, Welsh, LW, Bhavaraju-Sanka, R, Tobon Gonzalez, A, Jones, F, Saklad, A, Nations, S, Trivedi, J, Hopkins, S, Kazamel, M, Alsharabati, M, Lu, L, Mumfrey-Thomas, S, Woodall, A, Richman, D, Butters, J, Lindsay, M, Mozaffar, T, Cash, T, Goyal, N, Roy, G, Mathew, V, Maqsood, F, Minton, B, Jones, HJ, Rosenfeld, J, Garcia, R, Garcia, S, Echevarria, L, Pulley, M, Aranke, S, Berger, AR, Shah, J, Shabbir, Y, Smith, L, Varghese, M, Gutmann, L, Gutmann, L, Swenson, A, Olalde, H, Hafer-Macko, C, Kwan, J, Zilliox, L, Callison, K, DiSanzo, B, Naunton, K, Bilsker, M, Sharma, K, Reyes, E, Cooley, A, Michon, SC, Steele, J, Karam, CK, Chopra, M, Bird, S, Kaufman, J, Gallatti, N, Vu, T, Katzin, L, McClain, T, Harvey, B, Hart, A, Huynh, K, Beydoun, S, Chilingaryan, A, Droker, B, Lin, F, Shah, A, Tran, A, Akhter, S, Malekniazi, A, Tandan, R, Hehir, M, Waheed, W, Lucy, S, Weiss, M, Distad, J, Downing, S, Strom, S, Lisak, R, Bernitsas, E, Khan, O, Kumar Sriwastava, S, Tselis, A, Jia, K, Bertorini, T, Arnold, T, Henderson, K, Pillai, R, Liu, Y, Wheeler, L, Hewlett, J, Vanderhook, M, Dicapua, D, Keung, B, Kumar, A, Patwa, H, Robeson, K, Nye, J, Vu, H & REGAIN Study Group 2017, The Lancet Neurology, vol 16, no. 12, pp. 976-986. DOI: 10.1016/S1474-4422(17)30369-1

Introduction to supplement: the current status of treatment for ALS
Miller, RG & Appel, SH 2017, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 18, pp. 1-4. DOI: 10.1080/21678421.2017.1361447

Serum C-Reactive Protein as a Prognostic Biomarker in Amyotrophic Lateral Sclerosis
Lunetta, C, Lizio, A, Maestri, E, Sansone, VA, Mora, G, Miller, RG, Appel, SH & Chiò, A 2017, JAMA Neurology, vol 74, no. 6, pp. 660-667. DOI: 10.1001/jamaneurol.2016.6179

Pyrimethamine Significantly Lowers CSF/SOD1 in ALS Patients With SOD1 Mutations
Lange, DJ, Shahbazi, M, Silani, V, Ludolph, AC, Weishaupt, JH, Ajroud-Driss, S, Fields, KG, Remanan, R, Appel, SH, Morelli, C, Doretti, A, Maderna, L, Messina, S, Weiland, U, Marklund, SL & Andersen, PM 2017, Annals of Neurology. DOI: 10.1002/ana.24950

Clinical Significance of TDP-43 Neuropathology in Amyotrophic Lateral Sclerosis
Cykowski, MD, Powell, SZ, Peterson, LE, Appel, JW, Rivera, AL, Takei, H, Chang, E & Appel, SH 2017, Journal of Neuropathology and Experimental Neurology, vol 76, no. 5, pp. 402-413. DOI: 10.1093/jnen/nlx025

Characterization of Gene Expression Phenotype in Amyotrophic Lateral Sclerosis Monocytes
Zhao, W, Beers, DR, Hooten, KG, Sieglaff, DH, Zhang, A, Kalyana-Sundaram, S, Traini, CM, Halsey, WS, Hughes, AM, Sathe, GM, Livi, GP, Fan, GH & Appel, SH 2017, JAMA Neurology, vol 74, no. 6, pp. 677-685. DOI: 10.1001/jamaneurol.2017.0357

ALS patients' regulatory T lymphocytes are dysfunctional, and correlate with disease progression rate and severity
Beers, DR, Zhao, W, Wang, J, Zhang, X, Wen, S, Neal, D, Thonhoff, JR, Alsuliman, AS, Shpall, EJ, Rezvani, K & Appel, SH 2017, JCI insight, vol 2, no. 5, pp. e89530. DOI: 10.1172/jci.insight.89530

TDP-43 Depletion in Microglia Promotes Amyloid Clearance but Also Induces Synapse Loss
Paolicelli, RC, Jawaid, A, Henstridge, CM, Valeri, A, Merlini, M, Robinson, JL, Lee, EB, Rose, J, Appel, S, Lee, VMY, Trojanowski, JQ, Spires-Jones, T, Schulz, PE & Rajendran, L 2017, Neuron. DOI: 10.1016/j.neuron.2017.05.037

A robust, good manufacturing practice–compliant, clinical-scale procedure to generate regulatory T cells from patients with amyotrophic lateral sclerosis for adoptive cell therapy
Alsuliman, A, Appel, SH, Beers, DR, Basar, R, Shaim, H, Kaur, I, Zulovich, J, Yvon, E, Muftuoglu, M, Imahashi, N, Kondo, K, Liu, E, Shpall, EJ & Rezvani, K 2016, Cytotherapy, vol 18, no. 10, pp. 1312-1324. DOI: 10.1016/j.jcyt.2016.06.012

Stem cells in amyotrophic lateral sclerosis: Ready for prime time?
Appel, SH & Armon, C 2016, Neurology, vol 87, no. 4, pp. 348-349. DOI: 10.1212/WNL.0000000000002906

Defining SOD1 ALS natural history to guide therapeutic clinical trial design
Bali, T, Self, W, Liu, J, Siddique, T, Wang, LH, Bird, TD, Ratti, E, Atassi, N, Boylan, KB, Glass, JD, Maragakis, NJ, Caress, JB, McCluskey, LF, Appel, SH, Wymer, JP, Gibson, S, Zinman, L, Mozaffar, T, Callaghan, B, McVey, AL, Jockel-Balsarotti, J, Allred, P, Fisher, ER, Lopate, G, Pestronk, A, Cudkowicz, ME & Miller, TM 2016, Journal of Neurology, Neurosurgery and Psychiatry. DOI: 10.1136/jnnp-2016-313521

The immune system continues to knock at the ALS door
Staff, NP & Appel, SH 2016, Neuromuscular Disorders, vol 26, no. 6, pp. 335-336. DOI: 10.1016/j.nmd.2016.04.010

Amyotrophic lateral sclerosis
Simpson, EP & Appel, SH 2016, . in Neuroimmune Pharmacology. Springer International Publishing, pp. 493-502. DOI: 10.1007/978-3-319-44022-4_32

TDP-43 activates microglia through NF-?B and NLRP3 inflammasome
Zhao, W, Beers, DR, Bell, S, Wang, J, Wen, S, Baloh, RH & Appel, SH 2015, Experimental Neurology, vol 273, pp. 24-35. DOI: 10.1016/j.expneurol.2015.07.019