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Phenotypic characteristics of human bone marrow-derived endothelial progenitor cells in vitro support cell effectiveness for repair of the blood-spinal cord barrier in ALS.

Garbuzova-Davis S, Ehrhart J, Mustafa H, Llauget A, Boccio KJ, Sanberg PR, Appel SH, Borlongan CV.

Brain Res. 2019 Dec 1;1724:146428. doi: 10.1016/j.brainres.2019.146428. Epub 2019 Sep 4.


Dipeptide repeat (DPR) pathology in the skeletal muscle of ALS patients with C9ORF72 repeat expansion.

Cykowski MD, Dickson DW, Powell SZ, Arumanayagam AS, Rivera AL, Appel SH.

Acta Neuropathol. 2019 Oct;138(4):667-670. doi: 10.1007/s00401-019-02050-8. Epub 2019 Aug 2. No abstract available.


Multimodal 18F-AV-1451 and MRI Findings in Nonfluent Variant of Primary Progressive Aphasia: Possible Insights on Nodal Propagation of Tau Protein Across the Syntactic Network.

Pascual B, Funk Q, Zanotti-Fregonara P, Pal N, Rockers E, Yu M, Spann B, Román GC, Schulz PE, Karmonik C, Appel SH, Masdeu JC.

J Nucl Med. 2020 Feb;61(2):263-269. doi: 10.2967/jnumed.118.225508. Epub 2019 Jul 26.


Head-to-head comparison of 11C-PBR28 and 11C-ER176 for quantification of the translocator protein in the human brain.

Zanotti-Fregonara P, Pascual B, Veronese M, Yu M, Beers D, Appel SH, Masdeu JC.

Eur J Nucl Med Mol Imaging. 2019 Aug;46(9):1822-1829. doi: 10.1007/s00259-019-04349-w. Epub 2019 May 31.


Human Bone Marrow Endothelial Progenitor Cell Transplantation into Symptomatic ALS Mice Delays Disease Progression and Increases Motor Neuron Survival by Repairing Blood-Spinal Cord Barrier.

Garbuzova-Davis S, Kurien C, Haller E, Eve DJ, Navarro S, Steiner G, Mahendrasah A, Hailu S, Khatib M, Boccio KJ, Borlongan CV, Van Loveren HR, Appel SH, Sanberg PR.

Sci Rep. 2019 Mar 27;9(1):5280. doi: 10.1038/s41598-019-41747-4.


Immune dysregulation in amyotrophic lateral sclerosis: mechanisms and emerging therapies.

Beers DR, Appel SH.

Lancet Neurol. 2019 Feb;18(2):211-220. doi: 10.1016/S1474-4422(18)30394-6. Review.


Functional alterations of myeloid cells during the course of Alzheimer's disease.

Thome AD, Faridar A, Beers DR, Thonhoff JR, Zhao W, Wen S, Pascual B, Masdeu JC, Appel SH.

Mol Neurodegener. 2018 Nov 13;13(1):61. doi: 10.1186/s13024-018-0293-1.


Transplantation of human bone marrow stem cells into symptomatic ALS mice enhances structural and functional blood-spinal cord barrier repair.

Garbuzova-Davis S, Haller E, Navarro S, Besong TE, Boccio KJ, Hailu S, Khatib M, Sanberg PR, Appel SH, Borlongan CV.

Exp Neurol. 2018 Dec;310:33-47. doi: 10.1016/j.expneurol.2018.08.012. Epub 2018 Aug 30.


Neuroinflammatory mechanisms in amyotrophic lateral sclerosis pathogenesis.

Thonhoff JR, Simpson EP, Appel SH.

Curr Opin Neurol. 2018 Oct;31(5):635-639. doi: 10.1097/WCO.0000000000000599. Review.


Expanded autologous regulatory T-lymphocyte infusions in ALS: A phase I, first-in-human study.

Thonhoff JR, Beers DR, Zhao W, Pleitez M, Simpson EP, Berry JD, Cudkowicz ME, Appel SH.

Neurol Neuroimmunol Neuroinflamm. 2018 May 18;5(4):e465. doi: 10.1212/NXI.0000000000000465. eCollection 2018 Jul.


Phosphorylated TDP-43 (pTDP-43) aggregates in the axial skeletal muscle of patients with sporadic and familial amyotrophic lateral sclerosis.

Cykowski MD, Powell SZ, Appel JW, Arumanayagam AS, Rivera AL, Appel SH.

Acta Neuropathol Commun. 2018 Apr 13;6(1):28. doi: 10.1186/s40478-018-0528-y.


The Role of Regulatory T Lymphocytes in Amyotrophic Lateral Sclerosis.

Beers DR, Zhao W, Appel SH.

JAMA Neurol. 2018 Jun 1;75(6):656-658. doi: 10.1001/jamaneurol.2018.0043. No abstract available.


Head-to-Head Comparison of 11C-PBR28 and 18F-GE180 for Quantification of the Translocator Protein in the Human Brain.

Zanotti-Fregonara P, Pascual B, Rizzo G, Yu M, Pal N, Beers D, Carter R, Appel SH, Atassi N, Masdeu JC.

J Nucl Med. 2018 Aug;59(8):1260-1266. doi: 10.2967/jnumed.117.203109. Epub 2018 Jan 18.


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.

Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):242-249. doi: 10.1080/21678421.2017.1421666. Epub 2018 Jan 8.


Introduction to supplement: the current status of treatment for ALS.

Miller RG, Appel SH.

Amyotroph Lateral Scler Frontotemporal Degener. 2017 Oct;18(sup1):1-4. doi: 10.1080/21678421.2017.1361447.


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.

J Neuropathol Exp Neurol. 2017 May 1;76(5):402-413. doi: 10.1093/jnen/nlx025.


Pyrimethamine significantly lowers cerebrospinal fluid Cu/Zn superoxide dismutase in amyotrophic lateral sclerosis 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.

Ann Neurol. 2017 Jun;81(6):837-848. doi: 10.1002/ana.24950. Epub 2017 Jun 9.


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.

JAMA Neurol. 2017 Jun 1;74(6):677-685. doi: 10.1001/jamaneurol.2017.0357.


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.

JAMA Neurol. 2017 Jun 1;74(6):660-667. doi: 10.1001/jamaneurol.2016.6179.


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.

JCI Insight. 2017 Mar 9;2(5):e89530. doi: 10.1172/jci.insight.89530.


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.

Cytotherapy. 2016 Oct;18(10):1312-24. doi: 10.1016/j.jcyt.2016.06.012. Epub 2016 Aug 3.


Stem cells in amyotrophic lateral sclerosis: Ready for prime time?

Appel SH, Armon C.

Neurology. 2016 Jul 26;87(4):348-9. doi: 10.1212/WNL.0000000000002906. Epub 2016 Jun 29. No abstract available.


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.

J Neurol Neurosurg Psychiatry. 2017 Feb;88(2):99-105. doi: 10.1136/jnnp-2016-313521. Epub 2016 Jun 3.


The immune system continues to knock at the ALS door.

Staff NP, Appel SH.

Neuromuscul Disord. 2016 Jun;26(6):335-6. doi: 10.1016/j.nmd.2016.04.010. Epub 2016 Apr 19. No abstract available.


TDP-43 activates microglia through NF-κB and NLRP3 inflammasome.

Zhao W, Beers DR, Bell S, Wang J, Wen S, Baloh RH, Appel SH.

Exp Neurol. 2015 Nov;273:24-35. doi: 10.1016/j.expneurol.2015.07.019. Epub 2015 Jul 26.


Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS.

Miller RG, Block G, Katz JS, Barohn RJ, Gopalakrishnan V, Cudkowicz M, Zhang JR, McGrath MS, Ludington E, Appel SH, Azhir A; Phase 2 Trial NP001 Investigators.

Neurol Neuroimmunol Neuroinflamm. 2015 Apr 9;2(3):e100. doi: 10.1212/NXI.0000000000000100. eCollection 2015 Jun.


Protective and Toxic Neuroinflammation in Amyotrophic Lateral Sclerosis.

Hooten KG, Beers DR, Zhao W, Appel SH.

Neurotherapeutics. 2015 Apr;12(2):364-75. doi: 10.1007/s13311-014-0329-3. Review.


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.

Acta Neuropathol Commun. 2014 Dec 24;2:171. doi: 10.1186/s40478-014-0171-1.


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.

Curr Med Res Opin. 2014 Nov;30(11):2255-65. doi: 10.1185/03007995.2014.940040. Epub 2014 Jul 28.


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.

JAMA Neurol. 2014 Apr;71(4):449-53. doi: 10.1001/jamaneurol.2013.6237.


Special issue: A tribute to Jenny S. Henkel, Ph.D. (1955-2013).

Appel SH.

J Neuroimmune Pharmacol. 2013 Sep;8(4):757-1047. doi: 10.1007/s11481-013-9492-2. Epub 2013 Jul 31. No abstract available.


Immune-mediated mechanisms in the pathoprogression of amyotrophic lateral sclerosis.

Zhao W, Beers DR, Appel SH.

J Neuroimmune Pharmacol. 2013 Sep;8(4):888-99. doi: 10.1007/s11481-013-9489-x. Epub 2013 Jul 25. Review.


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.

Lancet Neurol. 2013 May;12(5):435-42. doi: 10.1016/S1474-4422(13)70061-9. Epub 2013 Mar 29. Erratum in: Lancet Neurol. 2013 May;12(5):423.


Regulatory T-lymphocytes mediate amyotrophic lateral sclerosis progression and survival.

Henkel JS, Beers DR, Wen S, Rivera AL, Toennis KM, Appel JE, Zhao W, Moore DH, Powell SZ, Appel SH.

EMBO Mol Med. 2013 Jan;5(1):64-79. doi: 10.1002/emmm.201201544. Epub 2012 Nov 9. Erratum in: EMBO Mol Med. 2013 Feb;5(2):326.


Amyotrophic lateral sclerosis, frontotemporal lobar dementia, and p62: a functional convergence?

Appel SH, Rowland LP.

Neurology. 2012 Oct 9;79(15):1526-7. doi: 10.1212/WNL.0b013e31826e26ec. Epub 2012 Sep 12. No abstract available.


Blood-spinal cord barrier breakdown and pericyte reductions in amyotrophic lateral sclerosis.

Winkler EA, Sengillo JD, Sullivan JS, Henkel JS, Appel SH, Zlokovic BV.

Acta Neuropathol. 2013 Jan;125(1):111-20. doi: 10.1007/s00401-012-1039-8. Epub 2012 Sep 1.


Regulatory T lymphocytes from ALS mice suppress microglia and effector T lymphocytes through different cytokine-mediated mechanisms.

Zhao W, Beers DR, Liao B, Henkel JS, Appel SH.

Neurobiol Dis. 2012 Dec;48(3):418-28. doi: 10.1016/j.nbd.2012.07.008. Epub 2012 Jul 17.


Inflammation in Parkinson's disease: cause or consequence?

Appel SH.

Mov Disord. 2012 Aug;27(9):1075-7. doi: 10.1002/mds.25111. Epub 2012 Jul 13. No abstract available.


Transformation from a neuroprotective to a neurotoxic microglial phenotype in a mouse model of ALS.

Liao B, Zhao W, Beers DR, Henkel JS, Appel SH.

Exp Neurol. 2012 Sep;237(1):147-52. doi: 10.1016/j.expneurol.2012.06.011. Epub 2012 Jun 23.


Peripheral nerve inflammation in ALS mice: cause or consequence.

Kano O, Beers DR, Henkel JS, Appel SH.

Neurology. 2012 Mar 13;78(11):833-5. doi: 10.1212/WNL.0b013e318249f776. Epub 2012 Feb 29. No abstract available.


Excess circulating alternatively activated myeloid (M2) cells accelerate ALS progression while inhibiting experimental autoimmune encephalomyelitis.

Vaknin I, Kunis G, Miller O, Butovsky O, Bukshpan S, Beers DR, Henkel JS, Yoles E, Appel SH, Schwartz M.

PLoS One. 2011;6(11):e26921. doi: 10.1371/journal.pone.0026921. Epub 2011 Nov 3.


Neuroprotective activities of regulatory T cells.

Gendelman HE, Appel SH.

Trends Mol Med. 2011 Dec;17(12):687-8. doi: 10.1016/j.molmed.2011.08.005. Epub 2011 Oct 11. No abstract available.


The microglial-motoneuron dialogue in ALS.

Appel SH, Zhao W, Beers DR, Henkel JS.

Acta Myol. 2011 Jun;30(1):4-8. Review.


Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: examining a more efficient trial design.

Miller RG, Moore DH, Forshew DA, Katz JS, Barohn RJ, Valan M, Bromberg MB, Goslin KL, Graves MC, McCluskey LF, McVey AL, Mozaffar T, Florence JM, Pestronk A, Ross M, Simpson EP, Appel SH; WALS Study Group.

Neurology. 2011 Sep 6;77(10):973-9. doi: 10.1212/WNL.0b013e31822dc7a5. Epub 2011 Aug 3.


Endogenous regulatory T lymphocytes ameliorate amyotrophic lateral sclerosis in mice and correlate with disease progression in patients with amyotrophic lateral sclerosis.

Beers DR, Henkel JS, Zhao W, Wang J, Huang A, Wen S, Liao B, Appel SH.

Brain. 2011 May;134(Pt 5):1293-314. doi: 10.1093/brain/awr074.


Neuroinflammation modulates distinct regional and temporal clinical responses in ALS mice.

Beers DR, Zhao W, Liao B, Kano O, Wang J, Huang A, Appel SH, Henkel JS.

Brain Behav Immun. 2011 Jul;25(5):1025-35. doi: 10.1016/j.bbi.2010.12.008. Epub 2010 Dec 19.


Trauma, TDP-43, and amyotrophic lateral sclerosis.

Appel SH, Cwik VA, Day JW.

Muscle Nerve. 2010 Dec;42(6):851-2. doi: 10.1002/mus.21939. No abstract available.


Does apolipoprotein E genotype modify the clinical expression of ALS?

Jawaid A, Poon M, Strutt AM, Rice LK, McDowell EJ, Salamone AR, Qureshi SU, Simpson E, Appel SH, York MK, Schulz PE.

Eur J Neurol. 2011 Apr;18(4):618-24. doi: 10.1111/j.1468-1331.2010.03225.x. Epub 2010 Sep 28.


ALS disease onset may occur later in patients with pre-morbid diabetes mellitus.

Jawaid A, Salamone AR, Strutt AM, Murthy SB, Wheaton M, McDowell EJ, Simpson E, Appel SH, York MK, Schulz PE.

Eur J Neurol. 2010 May;17(5):733-9. doi: 10.1111/j.1468-1331.2009.02923.x. Epub 2010 Jan 13.


T cell-microglial dialogue in Parkinson's disease and amyotrophic lateral sclerosis: are we listening?

Appel SH, Beers DR, Henkel JS.

Trends Immunol. 2010 Jan;31(1):7-17. doi: 10.1016/ Epub 2009 Oct 31. Review.

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