Target ALS

A New York based ALS research organization that's leading the fight for a cure.

Publications

A Snapshot of Published Collaborative Research

Target ALS-funded collaborations have been published in over 150 leading international journals, many of which are featured below.

  1. Abdeladim L, Matho KS, Clavreul S, Mahou P, Sintes JM, Solinas X, Arganda-Carreras I, Turney SG, Lichtman JW, Chessel A, Bemelmans AP, Loulier K, Supatto W, Livet J, Beaurepaire E. (2019). Multicolor multiscale brain imaging with chromatic multiphoton serial microscopy. Nat. Commun. 10, 1662.
  2. Alami NH, Smith RB, Carrasco MA, Williams LA, Winborn CS, Han SS, Kiskinis E, Winborn B, Freibaum BD, Kanagarj A, Clare AJ, Badders NM, Bilican B, Chaum E, Chandran S, Shaw CE, Eggan KC, Maniatis T, Taylor JP. (2014). Axonal transport of TDP-43 mRNA granules is impaired by ALS-causing mutations. Neuron. 81,536-553.
  3. Amoroso MW, Croft GF, Williams DJ, O’Keeffe S, Carrasco MA, Davis AR, Roybon L, Oakley DH, Maniatis T, Henderson CE, Wichterle H. (2013). Accelerated high-yield generation of limb-innervating motor neurons from human stem cells. J. Neurosci. 33,574-586.
  4. Antinone SE, Ghadge GD, Ostrow LW, Roos RP, Green WN. (2017). S-acylation of SOD1, CCS, and a stable SOD1-CCS heterodimer in human spinal cords from ALS and non-ALS subjects. Sci. Rep. 7,41141.
  5. Ayers JI, Diamond J, Sari A, Fromholt S, Galaleldeen A, Ostrow LW, Glass JD, Hart PJ, Borchelt DR. (2016). Distinct conformers of transmissible misfolded SOD1 distinguish human SOD1-FALS from other forms of familial and sporadic ALS. Acta Neuropathol. 132,827-840.
  6. Bączyk M, Alami NO, Delestrée N, Martinot C, Tang L, Commisso B, Bayer D, Doisne N, Frankel W, Manuel M, Roselli F, Zytnicki D. (2020). Synaptic Restoration by cAMP/PKA Drives Activity-Dependent Neuroprotection to Motoneurons in ALS. J. Exp. Med. 217,e20191734.
  7. Bakkar N, Kovalik T, Lorenzini I, Spangler S, Lacoste A, Sponaugle K, Ferrante P, Argentinis E, Sattler R, Bowser R. (2018). Artificial intelligence in neurodegenerative disease research: use of IBM Watson to identify additional RNA-binding proteins altered in amyotrophic lateral sclerosis. Acta Neuropathologica.135,227-247.
  8. Bakkar N, Kovalik T, Lorenzini I, Spangler S, Lacoste A, Sponaugle K, Ferrante P, Argentinis E, Sattler R, Bowser R. (2018). Artificial intelligence in neurodegenerative disease research: Use of IBM Watson to identify additional RNA binding proteins altered in amyotrophic lateral sclerosis. Acta Neuropathol 135: 227-247. PMID: 29134320
  9. Barmada SJ, Serio A, Arjun A, Bilican B, Daub A, Ando DM, Tsvetkov A, Pleiss M, Li X, Peisach D, Shaw C, Chandran S, Finkbeiner S. (2014). Autophagy induction enhances TDP43 turnover and survival in neuronal ALS models. Nat. Chem. Biol. 10,677-685.
  10. Batra R, Nelles DA, Pirie E, Blue SM, Marina RJ, Wang H, Chaim IA, Thomas JD, Zhang N, Nguyen V, Aigner S, Markmiller S, Xia G, Corbett KD, Swanson MS, Yeo GW. (2017). Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9. Cell 170,899-912.
  11. Becker LA, Huang B, Bieri G, Ma R, Knowles DA, Jafar-Nejad P, Messing J, Kim HJ, Soriano A, Auburger G, Pulst SM, Taylor JP, Rigo F, Gitler AD. (2017). Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice. Nature. 544,367-371.
  12. Benatar M, Zhang L, Wang L, Granit V, Statland J, Barohn R, Swenson A, Ravits J, Jackson C, Burns TM, Trivedi J, Pioro EP, Caress J, Katz J, McCauley JL, Rademakers R, Malaspina A, Ostrow LW, Wuu J; CReATe Consortium. (2020). Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS. Neurology.
  13. Bennett CL, Dastidar SG, Ling SC, Malik B, Ashe T, Wadhwa M, Miller DB, Lee C, Mitchell MB, van Es MA, Grunseich C, Chen Y, Sopher BL, Greensmith L, Cleveland DW, La Spada AR. (2018). Senataxin mutations elicit motor neuron degeneration phenotypes and yield TDP-43 mislocalization in ALS4 mice and human patients. Acta Neuropathol.136,425-443.
  14. Boehringer A, Garcia-Mansfield K, Singh G, Bakkar N, Pirrotte P, Bowser R (2017). ALS associated mutations in matrin 3 alter protein-protein interactions and impede mRNA nuclear export. Scientific reports.7,14529.
  15. Boehringer A, Garcia-Mansfield K, Singh G, Bakkar N, Pirrotte P, Bowser R. (2017). ALS associated mutations in Matrin 3 alter protein-protein interactions and impede mRNA nuclear export. Sci Rep, 7: 14529 doi:10.1038/s41598-017-14924-6. PMID: 29109432
  16. Boeynaems S, Bogaert E, Michiels E, Gijselinck I, Sieben A, Jovičić A, De Baets G, Scheveneels W1,2, Steyaert J, Cuijt I, Verstrepen KJ, Callaerts P, Rousseau F, Schymkowitz J, Cruts M, Van Broeckhoven C, Van Damme P, Gitler AD, Robberecht W, Van Den Bosch L. (2016) Drosophila screen connects nuclear transport genes to DPR pathology in c9ALS/FTD. Sci. Rep. 6,20877.
  17. Bogaert E, Boeynaems S, Kato M, Guo L, Caulfield TR, Steyaert J, Scheveneels W, Wilmans N, Haeck W, Hersmus N, Schymkowitz J, Rousseau F, Shorter J, Callaerts P, Robberecht W, Van Damme P, Van Den Bosch L. (2018). Molecular Dissection of FUS Points at Synergistic Effect of Low-Complexity Domains in Toxicity. Cell Rep. 24,529–537.
  18. Boulting GL, Kiskinis E, Croft GF, Amoroso MW, Oakley DH, Wainger BJ, Williams DJ, Kahler DJ, Yamaki M, Davidow L, Rodolfa CT, Dimos JT, Mikkilineni S, MacDermott AB, Woolf CJ, Henderson CE, Wichterle H, Eggan K. (2011). A functionally characterized test set of human induced pluripotent stem cells. Nat. Biotechnol. 29,279-286.
  19. Bush J, Aikawa H, Fuerst R, Li Y, Ursu A, Meyer S, Benhamou R, Chen J, Khan T, Wagner-Griffin S, Van Meter M, Tong Y, Olafson H, McKee K, Childs-Disney J, Gendron T, Zhang Y, Coyne A, Wang E, Yildirim I, Wang K, Petrucelli L, Rothstein J, Disney M. (2021). Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models. Science.
  20. Chai N, Haney MS, Couthouis J, Morgens DW, Benjamin A, Wu K, Ousey J, Fang S, Finer S, Bassik MC, Gitler AD. (2019). Genome-wide synthetic lethal CRISPR screen identifies FIS1 as a genetic interactor of ALS-linked C9ORF72. Brain Res.
  21. Cheng W, Wang S, Mestre AA, Fu C, Makarem A, Xian F, Hayes LR, Lopez-Gonzalez R, Drenner K, Jiang J, Cleveland DW, Sun S. (2018). C9ORF72 GGGGCC repeat-associated non-AUG translation is upregulated by stress through eIF2α phosphorylation. Nat. Commun. 9,51.
  22. Cheng W, Wang S, Zhang Z, Morgens DW, Hayes LR, Lee S, Portz B, Xie Y, Nguyen BV, Haney MS, Yan S, Dong D, Coyne AN, Yang J, Xian F, Cleveland DW, Qiu Z, Rothstein JD, Shorter J, Gao FB, Bassik MC, Sun S. (2019). CRISPR-Cas9 Screens Identify the RNA Helicase DDX3X as a Repressor of C9ORF72 (GGGGCC)n Repeat-Associated Non-AUG Translation. Neuron. 104,885-898.
  23. Chew J, Gendron TF, Prudencio M, Sasaguri H, Zhang YJ, Castanedes-Casey M, Lee CW, Jansen-West K, Kurti A, Murray ME, Bieniek KF, Bauer PO, Whitelaw EC, Rousseau L, Stankowski JN, Stetler C, Daughrity LD, Perkerson EA, Desaro P, Johnston A, Overstreet K3, Edbauer D, Rademakers R, Boylan KB, Dickson DW, Fryer JD, Petrucelli L. (2015). C9ORF72 repeat expansions in mice cause TDP-43 pathology, neuronal loss, and behavioral deficits. Science. 348,1151-1154.
  24. Chew J, Cook C, Gendron TF, Jansen-West K, Del Rosso G, Daughrity LM, Castanedes-Casey M, Kurti A, Stankowski JN, Disney MD, Rothstein JD, Dickson DW, Fryer JD, Zhang YJ, Petrucelli L. (2019). Aberrant deposition of stress granule-resident proteins linked to C9orf72-associated TDP-43 proteinopathy. Mol. Neurodegener. 14,9.
  25. Choi SY, Lopez-Gonzalez R, Krishnan G, Phillips HL, Li AN, Seeley WW, Yao WD, Almeida S, Gao FB. (2019). C9ORF72-ALS/FTD-associated poly(GR) binds Atp5a1 and compromises mitochondrial function in vivo. Nat. Neurosci.
  26. Cirulli ET, Lasseigne BN, Petrovski S, Sapp PC, Dion PA, Leblond CS, Couthouis J, Lu YF, 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 JM, Kenna KP, Smith BN, Topp S, Miller J, Gkazi A; FALS Sequencing Consortium, 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. (2015). Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways. Science. 347,1436-1441.
  27. Cleary JD, Ranum LP. (2013). Repeat-associated non-ATG (RAN) translation in neurological disease. Hum. Mol. Genet. 22,R45-51.
  28. Collins M, Li Y, Bowser R. (2020). RBM45 associates with nuclear stress bodies and forms nuclear inclusions during chronic stress and in neurodegenerative diseases. Acta Neuropathol Commun, Jun 26. 8:91. https://doi.org/10.1186/s40478-020-00965-y. PMID: 32586379
  29. Conlon EG, Fagegaltier D, Agius P, Davis-Porada J, Gregory J, Hubbard I, Kang K, Kim D; New York Genome Center ALS Consortium, Phatnani H, Kwan J, Sareen D, Broach JR, Simmons Z, Arcila-Londono X, Lee EB, Van Deerlin VM, Shneider NA, Fraenkel E, Ostrow LW, Baas F, Zaitlen N, Berry JD, Malaspina A, Fratta P, Cox GA, Thompson LM, Finkbeiner S, Dardiotis E, Miller TM, Chandran S, Pal S, Hornstein E, MacGowan DJ, Heiman-Patterson T, Hammell MG, Patsopoulos NA, Dubnau J, Nath A, Phatnani H, Shneider NA, Manley JL. (2018). Unexpected similarities between C9ORF72 and sporadic forms of ALS/FTD suggest a common disease mechanism. Elife. e37754.
  30. Couthouis J, Raphael AR, Daneshjou R, Gitler AD. (2014). Targeted exon capture and sequencing in sporadic amyotrophic lateral sclerosis. PLoS Genet. 10,e1004704.
  31. Coyne AN, Yamada SB, Siddegowda BB, Estes PS, Zaepfel BL, Johannesmeyer JS, Lockwood DB, Pham LT, Hart MP, Cassel JA, Freibaum B, Boehringer AV, Taylor JP, Reitz AB, Gitler AD, Zarnescu DC. (2015). Fragile X protein mitigates TDP-43 toxicity by remodeling RNA granules and restoring translation. Hum. Mol. Genet.
  32. Coyne AN, Zaepfel BL, Hayes L, Fitchman B, Salzberg Y, Luo EC, Bowen K, Trost H, Aigner S, Rigo F, Yeo GW, Harel A, Svendsen CN, Sareen D, Rothstein JD. (2020). G 4 C 2 Repeat RNA Initiates a POM121-Mediated Reduction in Specific Nucleoporins in C9orf72 ALS/FTD. Neuron. 20,30481-30485.
  33. de Boer AS, Koszka K, Kiskinis E, Suzuki N, Davis-Dusenbery BN, Eggan K. (2014). Genetic validation of a therapeutic target in a mouse model of ALS. Sci. Transl. Med. 6,248.
  34. Delestrée N, Manuel M, Igelsias C, Elbasiouny SM, Heckman CJ, Zytnicki D. (2014). Adult spinal motoneurones are not hyperexcitable in a mouse model of inherited amyotrophic lateral sclerosis. J. Physiol. 592,1687-1703.
  35. Donnelly CJ, Zhang PW, Pham JT, Heusler AR, Mistry NA, Vidensky S, Daley EL, Poth EM, Hoover B, Fines DM, Maragakis N, Tienari PJ, Petrucelli L, Traynor BJ, Wang J, Rigo F, Bennett CF, Blackshaw S, Sattler R, Rothstein JD. (2013). RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention. Neuron. 80,415-428.
  36. Dzhashiashvili Y, Monckton CP, Shah HS, Kunjamma RB, Popko B. (2019). The UPR-PERK pathway is not a promising therapeutic target for mutant SOD1-induced ALS. Neurobiol. Dis.
  37. Ebbert MTW, Farrugia SL, Sens JP, Jansen-West K, Gendron TF, Prudencio M, McLaughlin IJ, Bowman B, Seetin M, DeJesus-Hernandez M, Jackson J, Brown PH, Dickson DW, van Blitterswijk M, Rademakers R, Petrucelli L, Fryer JD. (2018). Long-read sequencing across the C9orf72 ‘GGGGCC’ repeat expansion: implications for clinical use and genetic discovery efforts in human disease. Mol. Neurodegener. 13,46.
  38. Figley MD, Bieri G, Kolaitis RM, Taylor JP, Gitler AD. (2014). Profilin 1 associates with stress granules and ALS-linked mutations alter stress granule dynamics. J Neurosci, 34,8083-8097.
  39. Figueroa-Romero C, Guo K, Murdock BJ, Paez-Colasante X, Bassis CM, ikhail KA, Raue KD, Evans MC, Taubman GF, McDermott AJ, O’Brien PD, Savelieff MG, Hur J, Feldman EL. (2019). Temporal evolution of the microbiome, immune system and epigenome with disease progression in ALS mice. Dis. Model Mech. 13,041947
  40. Foust KD, Salazar DL, Likhite S, Ferraiuolo L, Ditsworth D, Ilieva H, Meyer K, Schmelzer L, Braun L, Cleveland DW, Kaspar BK. (2013). Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS. Mol. Ther. 12,2148-2159
  41. Freibaum BD, Lu Y, Lopez-Gonzalez R, Kim NC, Almeida S, Lee KH, Badders N, Valentine M, Miller BL, Wong PC, Petrucelli L, Kim HJ, Gao FB, Taylor JP. (2015). Nature. 525,129-133.
  42. Gao FB, Richter JD, Cleveland DW. (2017). Rethinking unconventional translation in neurodegeneration. Cell. 171,994-1000.
  43. Gasset-Rosa F, Lu S, Yu H, Chen C, Melamed Z, Guo L, Shorter J, Da Cruz S, Cleveland DW. (2019). Cytoplasmic TDP-43 De-mixing Independent of Stress Granules Drives Inhibition of Nuclear Import, Loss of Nuclear TDP-43, and Cell Death. Neuron.
  44. Gates SN, Yokom AL, Lin J, Jackrel ME, Rizo AN, Kendsersky NM, Buell CE, Sweeny EA, Mack KL, Chuang E, Torrente MP, Su M, Shorter J, Southworth DR. (2017). Ratchet-like polypeptide translocation mechanism of the AAA+ disaggregase Hsp104. Science. 357,273-279.
  45. Gendron TF, van Blitterswijk M, Bieniek KF, Daughrity LM, Jiang J, Rush BK, Pedraza O, Lucas JA, Murray ME, Desaro P, Robertson A, Overstreet K, Thomas CS, Crook JE, Castanedes-Casey M, Rousseau L, Josephs KA, Parisi JE, Knopman DS, Petersen RC, Boeve BF, Graff-Radford NR, Rademakers R, Lagier-Tourenne C, Edbauer D, Cleveland DW, Dickson DW, Petrucelli L, Boylan KB. Cerebellar c9RAN proteins associate with clinical and neuropathological characteristics of C9ORF72 repeat expansion carriers. (2015). Acta Neuropathol. 130,559-573.
  46. Gendron TF, Chew J, Stankowski JN, Hayes LR, Zhang YJ, Prudencio M, Carlomagno Y, Daughrity LM, Jansen-West K, Perkerson EA, O’Raw A, Cook C, Pregent L, Belzil V, van Blitterswijk M, Tabassian LJ, Lee CW, Yue M, Tong J, Song Y, Castanedes-Casey M, Rousseau L, Phillips V, Dickson DW, Rademakers R, Fryer JD, Rush BK, Pedraza O, Caputo AM, Desaro P, Palmucci C, Robertson A, Heckman MG, Diehl NN, Wiggs E, Tierney M, Braun L, Farren J, Lacomis D, Ladha S, Fournier CN, McCluskey LF, Elman LB, Toledo JB, McBride JD, Tiloca C, Morelli C, Poletti B, Solca F, Prelle A, Wuu J, Jockel-Balsarotti J, Rigo F, Ambrose C, Datta A, Yang W, Raitcheva D, Antognetti G, McCampbell A, Van Swieten JC, Miller BL, Boxer AL, Brown RH, Bowser R, Miller TM, Trojanowski JQ, Grossman M, Berry JD, Hu WT, Ratti A, Traynor BJ, Disney MD, Benatar M, Silani V, Glass JD, Floeter MK, Rothstein JD, Boylan KB, Petrucelli L. (2017). Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis. Sci Transl Med. 9, 383.
  47. Ghadge GD, Sonobe Y, Camarena A, Drigotas C, Rigo F, Ling KK, Roos RP. (2019). Knockdown of GADD34 in neonatal mutant SOD1 mice ameliorates ALS. Neurobiol Dis
  48. Gomes E, Shorter J. (2018). The molecular language of membraneless organelles. J Biol Chem.
  49. Goodman LD, Prudencio M, Kramer NJ, Martinez-Ramirez LF, Srinivasan AR, Lan M, Parisi MJ, Zhu Y, Chew J, Cook CN, Berson A, Gitler AD, Petrucelli L, Bonini NM. (2019). Toxic expanded GGGGCC repeat transcription is mediated by the PAF1 complex in C9orf72-associated FTD. Nat. Neurosci. 22,863-874.
  50. Guo L, Kim HJ, Wang H, Monaghan J, Freyermuth F, Sung JC, O’Donovan K, Fare CM, Diaz Z, Singh N, Zhang ZC, Coughlin M, Sweeny EA, DeSantis ME, Jackrel ME, Rodell CB, Burdick JA, King OD, Gitler AD, Lagier-Tourenne C, Pandey UB, Chook YM, Taylor JP, Shorter J. (2018). Nuclear-Import Receptors Reverse Aberrant Phase Transitions of RNA-Binding Proteins with Prion-like Domains. Cell. 173,677-692.
  51. Haeusler AR, Donnelly CJ, Periz G, Simko EAJ, Shaw PG, Kim M, Maragakis NJ, Troncoso JC, Pandey A, Sattler R, Rothstein JD, Wang J. (2014). C9orf72 nucleotide repeat structures initiate molecular cascades of disease. Nature. 507,195-200.
  52. Haidet-Phillips AM, Hester ME, Miranda CJ, Meyer K, Braun L, Frakes A, Song S, Likhite S, Murtha MJ, Foust KD, Rao M, Eagle A, Kammesheidt A, Christensen A, Mendell JR, Burghes AH, Kaspar BK. (2011). Astrocytes from familial and sporadic ALS patients are toxic to motor neurons. Nat. Biotechnol. 29,824-828.
  53. Hasan, R., Humphrey, J., Bettencourt, C., Newcombe, J., NYGC ALS Consortium, Lashley, T., Fratta, P., & Raj, T. (2022). Transcriptomic analysis of frontotemporal lobar degeneration with TDP-43 pathology reveals cellular alterations across multiple brain regions. Acta neuropathologica, 143(3), 383–401. https://doi.org/10.1007/s00401-021-02399-9
  54. Henriques A, Croixmarie V, Priestman DA, Rosenbohm A, Dirrig-Grosch S, D’Ambra E, Huebecker M, Hussain G, Boursier-Neyret C, Echaniz-Laguna A, Ludolph AC, Platt FM, Walther B, Spedding M, Loeffler JP, Gonzalez de Aguilar JL. (2015). Amyotrophic lateral sclerosis and denervation alter sphingolipids and up-regulate glucosylceramide synthase. Hum. Mol. Genet. 24,7390-7405.
  55. Henriques A, Huebecker M, Blasco H, Keime C, Andres CR, Corcia P, Priestman DA, Platt FM, Spedding M, Loeffler JP. (2017). Inhibition of β-Glucocerebrosidase Activity Preserves Motor Unit Integrity in a Mouse Model of Amyotrophic Lateral Sclerosis. Sci. Rep. 7,5235.
  56. Huang X, Roet K, Zhang L, Lee S, Eggan K, Woolf C. (2021). Human amyotrophic lateral sclerosis excitability phenotype screen: target discovery and validation. Cell Rep.
  57. Ikiz B, Alvarez MJ, Ré DB, Le Verche V, Politi K, Lotti F, Phani S, Pradhan R, Yu C, Croft GF, Jacquier A, Henderson CE, Califano A, Przedborski S. (2015). The regulatory machinery of neurodegeneration in in vitro models of amyotrophic lateral sclerosis. Cell Rep. 12,335-345.
  58. Ishiura H, Takahashi Y, Goto J, Kost J, Gellera C, Gkazi AS, Miller J, Stockton J, Brooks WS, Boundy K, Polak M, Muñoz-Blanco JL, Esteban-Pérez J, Rábano A, Hardiman O, Morrison KE, Ticozzi N, Silani V, de Belleroche J, Glass JD, Kwok JB, Guillemin GJ, Chung RS, Tsuji S, Brown RH Jr, García-Redondo A, Rademakers R, Landers JE, Gitler AD, Rouleau GA, Cole NJ, Yerbury JJ, Atkin JD, Shaw CE, Nicholson GA, Blair IP. (2016). CCNF mutations in amyotrophic lateral sclerosis and frontotemporal dementia. Nat Commun. 7,11253.
  59. Jablonski AM, Lamitina T, Liachko NF, Sabatella M, Lu J, Zhang L, Ostrow LW, Gupta P, Wu C-Y, Doshi S, Mojsilovic-Petrovic J, Lans H, Wang J, Kraemer B, Kalb RG. (2015). Loss of RAD-23 Protects Against Models of Motor Neuron Disease by Enhancing Mutant Protein Clearance. J. Neurosci. 35,14286 –14306.
  60. Jackrel ME, DeSantis ME, Martinez BA, Castellano LM, Stewart RM, Caldwell KA, Caldwell GA, and Shorter J. (2014). Potentiated Hsp104 variants antagonize diverse proteotoxic misfolding events. Cell. 156,170-182.
  61. Jackrel ME and Shorter J. (2014). Reversing deleterious protein aggregation with re-engineered protein disaggregases. Cell Cycle. 13,1379-1383.
  62. Jackrel ME and Shorter J. (2014). Potentiated Hsp104 variants suppress toxicity of diverse neurodegenerative disease-linked proteins. Dis. Model Mech. 7,1175-1184
  63. Jackrel, ME, Tariq A, Yee K, Weitzman R, and Shorter J. (2014). Isolating potentiated Hsp104 variants using yeast proteinopathy models. J. Vis. Exp. 93,e52089.
  64. Jackrel ME, Yee K, Tariq A, Chen AI, Shorter J. (2015). Disparate Mutations Confer Therapeutic Gain of Hsp104 Function. ACS Chem. Biol. 10,2672-2679.
  65. Jiang J, Zhu Q, Gendron TF, Saberi S, McAlonis-Downes M, Seelman A, Stauffer JE, Jafar-Nejad P, Drenner K, Schulte D, Chun S, Sun S, Ling SC, Myers B, Engelhardt J, Katz M, Baughn M, Platoshyn O, Marsala M, Watt A, Heyser CJ, Ard MC, De Muynck L, Daughrity LM, Swing DA, Tessarollo L, Jung CJ, Delpoux A, Utzschneider DT, Hedrick SM, de Jong PJ, Edbauer D, Van Damme P, Petrucelli L, Shaw CE, Bennett CF, Da Cruz S, Ravits J, Rigo F, Cleveland DW, Lagier-Tourenne C. (2016). Neuron. 90,535-550.
  66. Jovičić A, Mertens J, Boeynaems S, Bogaert E, Chai N, Yamada SB, Paul JW 3rd, Sun S, Herdy JR, Bieri G, Kramer NJ, Gage FH, Van Den Bosch L, Robberecht W, Gitler AD. (2015). Modifiers of C9orf72 dipeptide repeat toxicity connect nucleocytoplasmic transport defects to FTD/ALS. Nat Neurosci. 18,1226-1229.
  67. Jovičić A, Paul JW 3rd, Gitler AD. (2016). Nuclear transport dysfunction: a common theme in amyotrophic lateral sclerosis and frontotemporal dementia. J. Neurochem. 138 Suppl 1:134-144.
  68. Kang SH, Fukaya M, Yang JK, Rothstein JD, Bergles DE. (2010). NG2+ CNS glial progenitors remain committed to the oligodendrocyte lineage in postnatal life and following neurodegeneration. Neuron. 68,668-681.
  69. Kang SH, Li Y, Fukaya M, Lorenzini I, Cleveland DW, Ostrow LW, Rothstein JD, Bergles DE. (2013). Degeneration and impaired regeneration of gray matter oligodendrocytes in amyotrophic lateral sclerosis. Nat. Neurosci. 16,571-579.
  70. Kaplan A, Spiller KJ, Towne C, Kanning KC, Choe GT, Geber A, Akay T, Aebischer P, Henderson CE. (2014). Neuronal matrix metalloproteinase-9 is a determinant of selective neurodegeneration. Neuron. 81,333-348.
  71. Kim H-J, Raphael AR, La Dow ES, McGurk L, Weber R, Trojanowski JQ, Lee VM-Y, Finkbeiner S, Gitler A, Bonini NM. Therapeutic modulation of eIF2 phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models. Nat. Genet. 46,152-160.
  72. Kim J, Hughes EG, Shetty AS, Arlotta P, Goff LA, Bergles DE, Brown SP. (2017). Changes in the excitability of neocortical neurons in a mouse model of amyotrophic lateral sclerosis are not specific to corticospinal neurons and are modulated by advancing disease. J Neurosci. 37,9037-9053
  73. Kiskinis E, Sandoe J, Williams LA, Boulting GL, Moccia R, Wainger BJ, Han S, Peng T, Thams S, Mikkilineni S, Mellin C, Merkle F, Ziller M, Oakley D, Ichida J, Dicostanza S, Atwater A, Maeder ML, Goodwin MJ, Nemesh J, Handsaker RE, Paull D, Noggle S, McCarroll SA, Joung JK, Woolf CJ, Brown RH, Eggan K. (2014). Pathways Disrupted in Human ALS Motor Neurons Identified Through Genetic Correction of Mutant SOD1. Cell Stem Cell. 14,781-795.
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