Publications


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:

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

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:

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:

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:

Protective and Toxic Neuroinflammation in Amyotrophic Lateral Sclerosis
Hooten, KG, Beers, DR, Zhao, W & Appel, SH 2015, Neurotherapeutics, vol 12, no. 2, pp. 364-375. DOI:

Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways
Cirulli, ET, Lasseigne, BN, Petrovski, S, Sapp, PC, Dion, PA, Leblond, CS, Couthouis, J, Lu, Y-F, Wang, Q, Krueger, BJ, Ren, Z, Keebler, J, Han, Y, Levy, SE, Boone, BE, Wimbish, JR, Waite, LL, Jones, AL, Carulli, JP, Day-Williams, AG, Staropoli, JF, Xin, WW, Chesi, A, Raphael, AR, McKenna-Yasek, D, Cady, J, Vianney de Jong, JMB, Kenna, KP, Smith, BN, Topp, S, Miller, J, Gkazi, A, Al-Chalabi, A, van den Berg, LH, Veldink, J, Silani, V, Ticozzi, N, Shaw, CE, Baloh, RH, Appel, S, Simpson, E, Lagier-Tourenne, C, Pulst, SM, Gibson, S, Trojanowski, JQ, Elman, L, McCluskey, L, Grossman, M, Shneider, NA, Chung, WK, Ravits, JM, Glass, JD, Sims, KB, Van Deerlin, VM, Maniatis, T, Hayes, SD, Ordureau, A, Swarup, S, Landers, J, Baas, F, Allen, AS, Bedlack, RS, Harper, JW, Gitler, AD, Rouleau, GA, Brown, R, Harms, MB, Cooper, GM, Harris, T, Myers, RM, Goldstein, DB & FALS Sequencing Consortium 2015, Science (New York, N.Y.), vol 347, no. 6229, pp. 1436-41. DOI:

The Role of Regulatory T Cells and Microglia in Amyotrophic Lateral Sclerosis
Beers, DR , Zhao, W, Hooten, KG & Appel, SH 2015, . in S David (ed.), Neuroinflammation: New Insights into Beneficial and Detrimental Functions. John Wiley & Sons, Inc. pp. 235-249. DOI:

Role of Inflammation in Neurodegenerative Diseases
Appel, SH , Beers, DR & Zhao, W 2014, . in Neurobiology of Brain Disorders: Biological Basis of Neurological and Psychiatric Disorders. Elsevier, pp. 380-395. DOI:

Neuroimmunology of Amyotrophic Lateral Sclerosis
Henkel, JS, Beers, DR, Zhao, W & Appel, SH 2014, . in Neuroinflammation and CNS Disorders. Wiley Blackwell, pp. 185-209. DOI:

Foreword
Appel, SH 2014, Unknown Journal, pp. vii-x. DOI:

TDP-43 pathology in the basal forebrain and hypothalamus of patients with amyotrophic lateral sclerosis
Cykowski, MD, Takei, H, Schulz, PE, Appel, SH & Powell, SZ 2014, Acta Neuropathologica Communications, vol 2, pp. 171. DOI:

Correction to: 'Seeking a measure of clinically meaningful change in ALS'
McElhiney, M, Rabkin, JG, Goetz, R, Katz, J, Miller, RG, Forshew, DA, David, W, Cudkowicz, M, Glass, JD, Appel, S, Simpson, E & Mitsumoto, H 2014, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 15, no. 7-8. DOI:

An open-label multicenter study to assess the safety of dextromethorphan/quinidine in patients with pseudobulbar affect associated with a range of underlying neurological conditions
Pattee, GL, Wymer, JP, Lomen-Hoerth, C, Appel, SH, Formella, AE & Pope, LE 2014, Current Medical Research and Opinion, vol 30, no. 11, pp. 2255-2265. DOI:

Seeking a measure of clinically meaningful change in ALS
Mcelhiney, M, Rabkin, JG, Goetz, R, Katz, J, Miller, RG, Forshew, DA, David, W, Cudkowicz, M, Glass, JD, Appel, S, Simpson, E & Mitsumoto, H 2014, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 15, no. 5-6, pp. 398-405. DOI:

TREM2 variant p.R47H as a risk factor for sporadic amyotrophic lateral sclerosis
Cady, J, Koval, ED, Benitez, BA, Zaidman, C, Jockel-Balsarotti, J, Allred, P, Baloh, RH, Ravits, J, Simpson, E, Appel, SH, Pestronk, A, Goate, AM, Miller, TM, Cruchaga, C & Harms, MB 2014, JAMA Neurology, vol 71, no. 4, pp. 449-453. DOI:

Immune-mediated mechanisms in the pathoprogression of amyotrophic lateral sclerosis
Zhao, W , Beers, DR & Appel, SH 2013, Journal of Neuroimmune Pharmacology, vol 8, no. 4, pp. 888-899. DOI:

A tribute to Jenny S. Henkel, Ph.D. (1955-2013)
Appel, SH 2013, Journal of Neuroimmune Pharmacology, vol 8, no. 4, pp. 757-1047. DOI:

Deciphering amyotrophic lateral sclerosis: What phenotype, neuropathology and genetics are telling us about pathogenesis
Ravits, J, Appel, S, Baloh, RH, Barohn, R, Rix Brooks, B, Elman, L, Floeter, MK, Henderson, C, Lomen-Hoerth, C, MacKlis, JD, McCluskey, L, Mitsumoto, H, Przedborski, S, Rothstein, J, Trojanowski, JQ, Van Den Berg, LH & Ringel, S 2013, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 14, no. SUPPL1, pp. 5-18. DOI:

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: A phase 1, randomised, first-in-man study
Miller, TM, Pestronk, A, David, W, Rothstein, J, Simpson, E, Appel, SH, Andres, PL, Mahoney, K, Allred, P, Alexander, K, Ostrow, LW, Schoenfeld, D, Macklin, EA, Norris, DA, Manousakis, G, Crisp, M, Smith, R, Bennett, CF, Bishop, KM & Cudkowicz, ME 2013, The Lancet Neurology, vol 12, no. 5, pp. 435-442. DOI: