jeff Rothstein

2025

Identification of a Subpopulation of Astrocyte Progenitor Cells in the Neonatal Subventricular Zone: Evidence that Migration is Regulated by Glutamate Signaling

Martinez-Lozada, Z., Guillem, A. M., Song, I., Gonzalez, M. V., Takano, H., Parikh, E., Rothstein, J. D., Putt, M. E. & Robinson, M. B., Feb 2025, In: Neurochemical Research. 50, 1, 77.

Research output: Contribution to journal › Article › peer-review

Open Access

2024

Apilimod dimesylate in C9orf72 amyotrophic lateral sclerosis: a randomized phase 2a clinical trial

Babu, S., Nicholson, K. A., Rothstein, J. D., Swenson, A., Sampognaro, P. J., Pant, P., Macklin, E. A., Spruill, S., Paganoni, S., Gendron, T. F., Prudencio, M., Petrucelli, L., Nix, D., Landrette, S., Nkrumah, E., Fandrick, K., Edwards, J. & Young, P. R., Sep 1 2024, In: Brain. 147, 9, p. 2998-3008 11 p.

Research output: Contribution to journal › Article › peer-review

A robust evaluation of TDP-43, poly GP, cellular pathology and behavior in an AAV-C9ORF72 (G₄C₂)₆₆ mouse model

Thompson, E. G., Spead, O., Akerman, S. C., Curcio, C., Zaepfel, B. L., Kent, E. R., Philips, T., Vijayakumar, B. G., Zacco, A., Zhou, W., Nagappan, G. & Rothstein, J. D., Dec 2024, In: Acta Neuropathologica Communications. 12, 1, 203.

Research output: Contribution to journal › Article › peer-review

Open Access

Disease related changes in ATAC-seq of iPSC-derived motor neuron lines from ALS patients and controls

the Answer ALS Consortium, Dec 2024, In: Nature communications. 15, 1, 3606.

Research output: Contribution to journal › Article › peer-review

Open Access

G₂C₄ targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons

Rothstein, J. D., Baskerville, V., Rapuri, S., Mehlhop, E., Jafar-Nejad, P., Rigo, F., Bennett, F., Mizielinska, S., Isaacs, A. & Coyne, A. N., Jun 2024, In: Acta neuropathologica. 147, 1, 1.

Research output: Contribution to journal › Article › peer-review

Inhibition of RNA splicing triggers CHMP7 nuclear entry, impacting TDP-43 function and leading to the onset of ALS cellular phenotypes

Al-Azzam, N., To, J. H., Gautam, V., Street, L. A., Nguyen, C. B., Naritomi, J. T., Lam, D. C., Madrigal, A. A., Lee, B., Jin, W., Avina, A., Mizrahi, O., Mueller, J. R., Ford, W., Schiavon, C. R., Rebollo, E., Vu, A. Q., Blue, S. M., Madakamutil, Y. L. & Manor, U. & 4 others, Rothstein, J. D., Coyne, A. N., Jovanovic, M. & Yeo, G. W., Dec 18 2024, In: Neuron. 112, 24, p. 4033-4047.e8

Research output: Contribution to journal › Article › peer-review

Open Access

Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis

Min, K., Yenilmez, B., Kelly, M., Echeverria, D., Elleby, M., Lifshitz, L. M., Raymond, N., Tsagkaraki, E., Harney, S. M., DiMarzio, C., Wang, H., McHugh, N., Bramato, B., Morrison, B., Rothstein, J. D., Khvorova, A. & Czech, M. P., Apr 2 2024, In: eLife. 12

Research output: Contribution to journal › Article › peer-review

Open Access

Mechanism-free repurposing of drugs for C9orf72-related ALS/FTD using large-scale genomic data

The International ALS Genomics Consortium, ITALSGEN Consortium, SLAGEN Consortium, Project MinE ALS Sequencing Consortium & The American Genome Center, Nov 13 2024, In: Cell Genomics. 4, 11, 100679.

Research output: Contribution to journal › Article › peer-review

Open Access

Monocarboxylate transporters facilitate succinate uptake into brown adipocytes

Reddy, A., Winther, S., Tran, N., Xiao, H., Jakob, J., Garrity, R., Smith, A., Ordonez, M., Laznik-Bogoslavski, D., Rothstein, J. D., Mills, E. L. & Chouchani, E. T., Mar 2024, In: Nature Metabolism. 6, 3, p. 567-577 11 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study

van den Berg, L. H., Rothstein, J. D., Shaw, P. J., Babu, S., Benatar, M., Bucelli, R. C., Genge, A., Glass, J. D., Hardiman, O., Libri, V., Mobach, T., Oskarsson, B., Pattee, G. L., Ravits, J., Shaw, C. E., Weber, M., Zinman, L., Jafar-nejad, P., Rigo, F. & Lin, L. & 9 others, Ferguson, T. A., Gotter, A. L., Graham, D., Monine, M., Inra, J., Sinks, S., Eraly, S., Garafalo, S. & Fradette, S., Sep 2024, In: The Lancet Neurology. 23, 9, p. 901-912 12 p.

Research output: Contribution to journal › Article › peer-review

2023

Genome-wide structural variant analysis identifies risk loci for non-Alzheimer's dementias

PROSPECT Consortium, The American Genome Center, International LBD Genomics Consortium & International ALS/FTD Consortium, Jun 14 2023, In: Cell Genomics. 3, 6, 100316.

Research output: Contribution to journal › Article › peer-review

Open Access

Large-scale differentiation of iPSC-derived motor neurons from ALS and control subjects

the Answer ALS Consortium, Apr 19 2023, In: Neuron. 111, 8, p. 1191-1204.e5

Research output: Contribution to journal › Article › peer-review

Open Access

NeuroLINCS Proteomics: Defining human-derived iPSC proteomes and protein signatures of pluripotency

NIH NeuroLINCS Consortium, Dec 2023, In: Scientific Data. 10, 1, 24.

Research output: Contribution to journal › Article › peer-review

Open Access

2022

Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines

Baxi, E. G., Thompson, T., Li, J., Kaye, J. A., Lim, R. G., Wu, J., Ramamoorthy, D., Lima, L., Vaibhav, V., Matlock, A., Frank, A., Coyne, A. N., Landin, B., Ornelas, L., Mosmiller, E., Thrower, S., Farr, S. M., Panther, L., Gomez, E. & Galvez, E. & 81 others, Perez, D., Meepe, I., Lei, S., Mandefro, B., Trost, H., Pinedo, L., Banuelos, M. G., Liu, C., Moran, R., Garcia, V., Workman, M., Ho, R., Wyman, S., Roggenbuck, J., Harms, M. B., Stocksdale, J., Miramontes, R., Wang, K., Venkatraman, V., Holewenski, R., Sundararaman, N., Pandey, R., Manalo, D. M., Donde, A., Huynh, N., Adam, M., Wassie, B. T., Vertudes, E., Amirani, N., Raja, K., Thomas, R., Hayes, L., Lenail, A., Cerezo, A., Luppino, S., Farrar, A., Pothier, L., Prina, C., Morgan, T., Jamil, A., Heintzman, S., Jockel-Balsarotti, J., Karanja, E., Markway, J., McCallum, M., Joslin, B., Alibazoglu, D., Kolb, S., Ajroud-Driss, S., Baloh, R., Heitzman, D., Miller, T., Glass, J. D., Patel-Murray, N. L., Yu, H., Sinani, E., Vigneswaran, P., Sherman, A. V., Ahmad, O., Roy, P., Beavers, J. C., Zeiler, S., Krakauer, J. W., Agurto, C., Cecchi, G., Bellard, M., Raghav, Y., Sachs, K., Ehrenberger, T., Bruce, E., Cudkowicz, M. E., Maragakis, N., Norel, R., Van Eyk, J. E., Finkbeiner, S., Berry, J., Sareen, D., Thompson, L. M., Fraenkel, E., Svendsen, C. N. & Rothstein, J. D., Feb 2022, In: Nature neuroscience. 25, 2, p. 226-237 12 p.

Research output: Contribution to journal › Article › peer-review

Open Access

A Phase 1 study of GDC-0134, a dual leucine zipper kinase inhibitor, in ALS

Katz, J. S., Rothstein, J. D., Cudkowicz, M. E., Genge, A., Oskarsson, B., Hains, A. B., Chen, C., Galanter, J., Burgess, B. L., Cho, W., Kerchner, G. A., Yeh, F. L., Ghosh, A. S., Cheeti, S., Brooks, L., Honigberg, L., Couch, J. A., Rothenberg, M. E., Brunstein, F. & Sharma, K. R. & 3 others, van den Berg, L., Berry, J. D. & Glass, J. D., Jan 2022, In: Annals of Clinical and Translational Neurology. 9, 1, p. 50-66 17 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial

Paganoni, S., Hendrix, S., Dickson, S. P., Knowlton, N., Berry, J. D., Elliott, M. A., Maiser, S., Karam, C., Caress, J. B., Owegi, M. A., Quick, A., Wymer, J., Goutman, S. A., Heitzman, D., Heiman-Patterson, T. D., Jackson, C., Quinn, C., Rothstein, J. D., Kasarskis, E. J. & Katz, J. & 30 others, Jenkins, L., Ladha, S. S., Miller, T. M., Scelsa, S. N., Vu, T. H., Fournier, C., Johnson, K. M., Swenson, A., Goyal, N., Pattee, G. L., Babu, S., Chase, M., Dagostino, D., Hall, M., Kittle, G., Eydinov, M., Ostrow, J., Pothier, L., Randall, R., Shefner, J. M., Sherman, A. V., Tustison, E., Vigneswaran, P., Yu, H., Cohen, J., Klee, J., Tanzi, R., Gilbert, W., Yeramian, P. & Cudkowicz, M., Aug 1 2022, In: Journal of Neurology, Neurosurgery and Psychiatry. 93, 8, p. 871-875 5 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Identification of Therapeutic Targets for Amyotrophic Lateral Sclerosis Using PandaOmics – An AI-Enabled Biological Target Discovery Platform

Pun, F. W., Liu, B. H. M., Long, X., Leung, H. W., Leung, G. H. D., Mewborne, Q. T., Gao, J., Shneyderman, A., Ozerov, I. V., Wang, J., Ren, F., Aliper, A., Bischof, E., Izumchenko, E., Guan, X., Zhang, K., Lu, B., Rothstein, J. D., Cudkowicz, M. E. & Zhavoronkov, A., Jun 28 2022, In: Frontiers in Aging Neuroscience. 14, 914017.

Research output: Contribution to journal › Article › peer-review

Open Access

Identifying patterns in amyotrophic lateral sclerosis progression from sparse longitudinal data

Answer ALS, Pooled Resource Open-Access ALS Clinical Trials Consortium & ALS/MND Natural History Consortium, Sep 2022, In: Nature Computational Science. 2, 9, p. 605-616 12 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Nuclear RNA binding regulates TDP-43 nuclear localization and passive nuclear export

Duan, L., Zaepfel, B. L., Aksenova, V., Dasso, M., Rothstein, J. D., Kalab, P. & Hayes, L. R., Jul 19 2022, In: Cell Reports. 40, 3, 111106.

Research output: Contribution to journal › Article › peer-review

Open Access

Poly(ADP-ribose) promotes toxicity of C9ORF72 arginine-rich dipeptide repeat proteins

Gao, J., Mewborne, Q. T., Girdhar, A., Sheth, U., Coyne, A. N., Punathil, R., Kang, B. G., Dasovich, M., Veire, A., DeJesus Hernandez, M., Liu, S., Shi, Z., Dafinca, R., Fouquerel, E., Talbot, K., Kam, T. I., Zhang, Y. J., Dickson, D., Petrucelli, L. & van Blitterswijk, M. & 8 others, Guo, L., Dawson, T. M., Dawson, V. L., Leung, A. K. L., Lloyd, T. E., Gendron, T. F., Rothstein, J. D. & Zhang, K., Sep 14 2022, In: Science translational medicine. 14, 662, abq3215.

Research output: Contribution to journal › Article › peer-review

Open Access

2021

A Helicase Unwinds Hexanucleotide Repeat RNA G-Quadruplexes and Facilitates Repeat-Associated Non-AUG Translation

Liu, H., Lu, Y. N., Paul, T., Periz, G., Banco, M. T., Ferré-D'Amaré, A. R., Rothstein, J. D., Hayes, L. R., Myong, S. & Wang, J., May 19 2021, In: Journal of the American Chemical Society. 143, 19, p. 7368-7379 12 p.

Research output: Contribution to journal › Article › peer-review

Open Access

ALSUntangled 55: vitamin E (α-tocopherol)

THE ALSUNTANGLED GROUP, 2021, In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 22, 1-2, p. 154-160 7 p.

Research output: Contribution to journal › Article › peer-review

An integrated multi-omic analysis of iPSC-derived motor neurons from C9ORF72 ALS patients

The NeuroLINCS Consortium & NYGC ALS Consortium, Nov 19 2021, In: iScience. 24, 11, 103221.

Research output: Contribution to journal › Article › peer-review

Open Access

Association of Variants in the SPTLC1 Gene with Juvenile Amyotrophic Lateral Sclerosis

Johnson, J. O., Chia, R., Miller, D. E., Li, R., Kumaran, R., Abramzon, Y., Alahmady, N., Renton, A. E., Topp, S. D., Gibbs, J. R., Cookson, M. R., Sabir, M. S., Dalgard, C. L., Troakes, C., Jones, A. R., Shatunov, A., Iacoangeli, A., Al Khleifat, A., Ticozzi, N. & Silani, V. & 267 others, Gellera, C., Blair, I. P., Dobson-Stone, C., Kwok, J. B., Bonkowski, E. S., Palvadeau, R., Tienari, P. J., Morrison, K. E., Shaw, P. J., Al-Chalabi, A., Brown, R. H., Calvo, A., Mora, G., Al-Saif, H., Gotkine, M., Leigh, F., Chang, I. J., Perlman, S. J., Glass, I., Scott, A. I., Shaw, C. E., Basak, A. N., Landers, J. E., Chiò, A., Crawford, T. O., Smith, B. N., Traynor, B. J., Smith, B. N., Fallini, C., Gkazi, A. S., Topp, S. D., Scotter, E. L., Kenna, K. P., Keagle, P., Tiloca, C., Vance, C., Troakes, C., Colombrita, C., King, A., Pensato, V., Castellotti, B., Baas, F., Ten Asbroek, A. L. M. A., McKenna-Yasek, D., McLaughlin, R. L., Polak, M., Asress, S., Esteban-Pérez, J., Stevic, Z., D'Alfonso, S., Mazzini, L., Comi, G. P., Del Bo, R., Ceroni, M., Gagliardi, S., Querin, G., Bertolin, C., Van Rheenen, W., Rademakers, R., Van Blitterswijk, M., Lauria, G., Duga, S., Corti, S., Cereda, C., Corrado, L., Sorarù, G., Williams, K. L., Nicholson, G. A., Blair, I. P., Leblond-Manry, C., Rouleau, G. A., Hardiman, O., Morrison, K. E., Veldink, J. H., Van Den Berg, L. H., Pall, H., Turner, M. R., Talbot, K., Taroni, F., García-Redondo, A., Wu, Z., Glass, J. D., Gellera, C., Ratti, A., Brown, R. H., Landers, J. E., Dalgard, C. L., Adeleye, A., Soltis, A. R., Alba, C., Viollet, C., Bacikova, D., Hupalo, D. N., Sukumar, G., Pollard, H. B., Wilkerson, M. D., Martinez, E. M. G., Abramzon, Y., Ahmed, S., Arepalli, S., Baloh, R. H., Bowser, R., Brady, C. B., Brice, A., Broach, J., Campbell, R. H., Camu, W., Cooper-Knock, J., Ding, J., Drepper, C., Drory, V. E., Dunckley, T. L., Eicher, J. D., England, B. K., Faghri, F., Feldman, E., Floeter, M. K., Fratta, P., Geiger, J. T., Gerhard, G., Gibson, S. B., Glass, J. D., Hardy, J., Harms, M. B., Heiman-Patterson, T. D., Hernandez, D. G., Jansson, L., Kirby, J., Kowall, N. W., Laaksovirta, H., Landeck, N., Landi, F., Le Ber, I., Lumbroso, S., Macgowan, D. J. L., Maragakis, N. J., Mouzat, K., Murphy, N. A., Myllykangas, L., Nalls, M. A., Orrell, R. W., Ostrow, L. W., Pamphlett, R., Pickering-Brown, S., Pioro, E. P., Pletnikova, O., Pliner, H. A., Pulst, S. M., Ravits, J. M., Renton, A. E., Rivera, A., Robberecht, W., Rogaeva, E., Rollinson, S., Rothstein, J. D., Scholz, S. W., Sendtner, M., Sidle, K. C., Simmons, Z., Singleton, A. B., Smith, N., Stone, D. J., Tienari, P. J., Troncoso, J. C., Valori, M., Van Damme, P., Van Deerlin, V. M., Van Den Bosch, L., Zinman, L., Landers, J. E., Chiò, A., Traynor, B. J., Angelocola, S. M., Ausiello, F. P., Barberis, M., Bartolomei, I., Battistini, S., Bersano, E., Bisogni, G., Borghero, G., Brunetti, M., Cabona, C., Calvo, A., Canale, F., Canosa, A., Cantisani, T. A., Capasso, M., Caponnetto, C., Cardinali, P., Carrera, P., Casale, F., Chiò, A., Colletti, T., Conforti, F. L., Conte, A., Conti, E., Corbo, M., Cuccu, S., Dalla Bella, E., D'Errico, E., Demarco, G., Dubbioso, R., Ferrarese, C., Ferraro, P. M., Filippi, M., Fini, N., Floris, G., Fuda, G., Gallone, S., Gianferrari, G., Giannini, F., Grassano, M., Greco, L., Iazzolino, B., Introna, A., La Bella, V., Lattante, S., Liguori, R., Logroscino, G., Logullo, F. O., Lunetta, C., Mandich, P., Mandrioli, J., Manera, U., Manganelli, F., Marangi, G., Marinou, K., Marrosu, M. G., Martinelli, I., Messina, S., Moglia, C., Mosca, L., Murru, M. R., Origone, P., Passaniti, C., Petrelli, C., Petrucci, A., Pozzi, S., Pugliatti, M., Quattrini, A., Ricci, C., Riolo, G., Riva, N., Russo, M., Sabatelli, M., Salamone, P., Salivetto, M., Salvi, F., Santarelli, M., Sbaiz, L., Sideri, R., Simone, I., Simonini, C., Spataro, R., Tanel, R., Tedeschi, G., Ticca, A., Torriello, A., Tranquilli, S., Tremolizzo, L., Trojsi, F., Vasta, R., Vacchiano, V., Vita, G., Volanti, P., Zollino, M. & Zucchi, E., Oct 1 2021, In: JAMA Neurology. 78, 10, p. 1236-1248 13 p.

Research output: Contribution to journal › Article › peer-review

Open Access

Increased synthesis of pro-inflammatory cytokines in C9ORF72 patients

Pinilla, G., Kumar, A., Floaters, M. K., Pardo, C. A., Rothstein, J. & Ilieva, H., 2021, In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 22, 7-8, p. 517-527 11 p.

Research output: Contribution to journal › Article › peer-review

Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis

Paganoni, S., Hendrix, S., Dickson, S. P., Knowlton, N., Macklin, E. A., Berry, J. D., Elliott, M. A., Maiser, S., Karam, C., Caress, J. B., Owegi, M. A., Quick, A., Wymer, J., Goutman, S. A., Heitzman, D., Heiman-Patterson, T. D., Jackson, C. E., Quinn, C., Rothstein, J. D. & Kasarskis, E. J. & 41 others, Katz, J., Jenkins, L., Ladha, S., Miller, T. M., Scelsa, S. N., Vu, T. H., Fournier, C. N., Glass, J. D., Johnson, K. M., Swenson, A., Goyal, N. A., Pattee, G. L., Andres, P. L., Babu, S., Chase, M., Dagostino, D., Hall, M., Kittle, G., Eydinov, M., McGovern, M., Ostrow, J., Pothier, L., Randall, R., Shefner, J. M., Sherman, A. V., St Pierre, M. E., Tustison, E., Vigneswaran, P., Walker, J., Yu, H., Chan, J., Wittes, J., Yu, Z. F., Cohen, J., Klee, J., Leslie, K., Tanzi, R. E., Gilbert, W., Yeramian, P. D., Schoenfeld, D. & Cudkowicz, M. E., Jan 2021, In: Muscle and Nerve. 63, 1, p. 31-39 9 p.

Research output: Contribution to journal › Article › peer-review

Open Access

5 Scopus citations

Macrophage monocarboxylate transporter 1 promotes peripheral nerve regeneration after injury in mice

Jha, M. K., Passero, J. V., Rawat, A., Ament, X. H., Yang, F., Vidensky, S., Collins, S. L., Horton, M. R., Hoke, A., Rutter, G. A., Latremoliere, A., Rothstein, J. D. & Morrison, B. M., Nov 2021, In: Journal of Clinical Investigation. 131, 21, e141964.

Research output: Contribution to journal › Article › peer-review

Open Access

MC-100093, a novel β-lactam glutamate transporter-1 enhancer devoid of antimicrobial properties, attenuates cocaine relapse in rats

Knackstedt, L. A., Wu, L., Rothstein, J., Vidensky, S., Gordon, J., Ramanjulu, M., Dunman, P., Blass, B., Childers, W. & Abou-Gharbia, M., Aug 1 2021, In: Journal of Pharmacology and Experimental Therapeutics. 378, 2, p. 51-59 9 p.

Research output: Contribution to journal › Article › peer-review

Open Access

MCT1 Deletion in Oligodendrocyte Lineage Cells Causes Late-Onset Hypomyelination and Axonal Degeneration

Philips, T., Mironova, Y. A., Jouroukhin, Y., Chew, J., Vidensky, S., Farah, M. H., Pletnikov, M. V., Bergles, D. E., Morrison, B. M. & Rothstein, J. D., Jan 12 2021, In: Cell Reports. 34, 2, 108610.

Research output: Contribution to journal › Article › peer-review

Open Access

7 Scopus citations

Metabolic support of tumour-infiltrating regulatory T cells by lactic acid

Watson, M. L. J., Vignali, P. D. A., Mullett, S. J., Overacre-Delgoffe, A. E., Peralta, R. M., Grebinoski, S., Menk, A. V., Rittenhouse, N. L., DePeaux, K., Whetstone, R. D., Vignali, D. A. A., Hand, T. W., Poholek, A. C., Morrison, B. M., Rothstein, J. D., Wendell, S. G. & Delgoffe, G. M., Mar 25 2021, In: Nature. 591, 7851, p. 645-651 7 p.

Research output: Contribution to journal › Article › peer-review

Open Access

11 Scopus citations

Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS

Coyne, A. N., Baskerville, V., Zaepfel, B. L., Dickson, D. W., Rigo, F., Bennett, F., Patrick Lusk, C. & Rothstein, J. D., Jul 28 2021, In: Science translational medicine. 13, 604, eabe1923.

Research output: Contribution to journal › Article › peer-review

Open Access

Nuclear export and translation of circular repeat-containing intronic RNA in C9ORF72-ALS/FTD

Wang, S., Latallo, M. J., Zhang, Z., Huang, B., Bobrovnikov, D. G., Dong, D., Livingston, N. M., Tjoeng, W., Hayes, L. R., Rothstein, J. D., Ostrow, L. W., Wu, B. & Sun, S., Dec 1 2021, In: Nature communications. 12, 1, 4908.

Research output: Contribution to journal › Article › peer-review

Open Access

Nuclear lamina invaginations are not a pathological feature of C9orf72 ALS/FTD

Coyne, A. N. & Rothstein, J. D., Dec 2021, In: Acta Neuropathologica Communications. 9, 1, 45.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Nuclei isolation and super-resolution structured illumination microscopy for examining nucleoporin alterations in human neurodegeneration

Coyne, A. N. & Rothstein, J. D., Sep 2021, In: Journal of Visualized Experiments. 2021, 175, e62789.

Research output: Contribution to journal › Article › peer-review

Pathogenic Huntingtin Repeat Expansions in Patients with Frontotemporal Dementia and Amyotrophic Lateral Sclerosis

The PROSPECT Consortium, The American Genome Center (TAGC), The FALS Sequencing Consortium, The Genomics England Research Consortium, The International ALS/FTD Genomics Consortium (iAFGC), The International FTD Genetics Consortium (IFGC), The International LBD Genomics Consortium (iLBDGC), The NYGC ALS Consortium & the University of Maryland Brain and Tissue Bank and NIH NeuroBioBank, Feb 3 2021, In: Neuron. 109, 3, p. 448-460.e4

Research output: Contribution to journal › Article › peer-review

Open Access

3 Scopus citations

Polyadenylated RNA and RNA-Binding Proteins Exhibit Unique Response to Hyperosmotic Stress

Zaepfel, B. L. & Rothstein, J. D., Dec 14 2021, In: Frontiers in Cell and Developmental Biology. 9, 809859.

Research output: Contribution to journal › Article › peer-review

Open Access

Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G₄C₂) repeat expansion in vitro and in vivo ALS models

Bush, J. A., Aikawa, H., Fuerst, R., Li, Y., Ursu, A., Meyer, S. M., Benhamou, R. I., Chen, J. L., Khan, T., Wagner-Griffin, S., van Meter, M. J., Tong, Y., Olafson, H., McKee, K. K., Childs-Disney, J. L., Gendron, T. F., Zhang, Y., Coyne, A. N., Wang, E. T. & Yildirim, I. & 4 others, Wang, K. W., Petrucelli, L., Rothstein, J. D. & Disney, M. D., Oct 27 2021, In: Science translational medicine. 13, 617, eabd5991.

Research output: Contribution to journal › Article › peer-review

Open Access

The ESCRT-III protein VPS4, but not CHMP4B or CHMP2B, is pathologically increased in familial and sporadic ALS neuronal nuclei

Coyne, A. N. & Rothstein, J. D., Dec 2021, In: Acta Neuropathologica Communications. 9, 1, 127.

Research output: Contribution to journal › Article › peer-review

Open Access

UPF1 reduces C9orf72 HRE-induced neurotoxicity in the absence of nonsense-mediated decay dysfunction

Zaepfel, B. L., Zhang, Z., Maulding, K., Coyne, A. N., Cheng, W., Hayes, L. R., Lloyd, T. E., Sun, S. & Rothstein, J. D., Mar 30 2021, In: Cell Reports. 34, 13, 108925.

Research output: Contribution to journal › Article › peer-review

Open Access

2020

Absence of Survival and Motor Deficits in 500 Repeat C9ORF72 BAC Mice

Mordes, D. A., Morrison, B. M., Ament, X. H., Cantrell, C., Mok, J., Eggan, P., Xue, C., Wang, J. Y., Eggan, K. & Rothstein, J. D., Nov 25 2020, In: Neuron. 108, 4, p. 775-783.e4

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Amyotrophic lateral sclerosis care and research in the United States during the COVID-19 pandemic: Challenges and opportunities

Andrews, J. A., Berry, J. D., Baloh, R. H., Carberry, N., Cudkowicz, M. E., Dedi, B., Glass, J., Maragakis, N. J., Miller, T. M., Paganoni, S., Rothstein, J. D., Shefner, J. M., Simmons, Z., Weiss, M. D. & Bedlack, R. S., Aug 1 2020, In: Muscle and Nerve. 62, 2, p. 182-186 5 p.

Research output: Contribution to journal › Article › peer-review

Open Access

13 Scopus citations

Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model

Nguyen, L., Montrasio, F., Pattamatta, A., Tusi, S. K., Bardhi, O., Meyer, K. D., Hayes, L., Nakamura, K., Banez-Coronel, M., Coyne, A., Guo, S., Laboissonniere, L. A., Gu, Y., Narayanan, S., Smith, B., Nitsch, R. M., Kankel, M. W., Rushe, M., Rothstein, J. & Zu, T. & 2 others, Grimm, J. & Ranum, L. P. W., Feb 19 2020, In: Neuron. 105, 4, p. 645-662.e11

Research output: Contribution to journal › Article › peer-review

Open Access

17 Scopus citations

Astrocyte Diversity: Current Insights and Future Directions

Westergard, T. & Rothstein, J. D., Jun 1 2020, In: Neurochemical Research. 45, 6, p. 1298-1305 8 p.

Research output: Contribution to journal › Article › peer-review

9 Scopus citations

C9orf72 arginine-rich dipeptide repeat proteins disrupt karyopherin-mediated nuclear import

Hayes, L. R., Duan, L., Bowen, K., Kalab, P. & Rothstein, J. D., Mar 2020, In: eLife. 9, e51685.

Research output: Contribution to journal › Article › peer-review

Open Access

21 Scopus citations

G₄C₂ Repeat RNA Initiates a POM121-Mediated Reduction in Specific Nucleoporins in C9orf72 ALS/FTD

Coyne, A. N., Zaepfel, B. L., Hayes, L., Fitchman, B., Salzberg, Y., Luo, E. C., Bowen, K., Trost, H., Aigner, S., Rigo, F., Yeo, G. W., Harel, A., Svendsen, C. N., Sareen, D. & Rothstein, J. D., Sep 23 2020, In: Neuron. 107, 6, p. 1124-1140.e11

Research output: Contribution to journal › Article › peer-review

Open Access

10 Scopus citations

Hydrogen peroxide triggers an increase in cell surface expression of system x_c ⁻ in cultured human glioma cells

Chase, L. A., VerHeulen Kleyn, M., Schiller, N. T., King, A. G., Flores, G., Engelsman, S. B., Bowles, C., Smith, S. L., Robinson, A. E. & Rothstein, J., Mar 2020, In: Neurochemistry International. 134, 104648.

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Monocarboxylate transporter 1 in Schwann cells contributes to maintenance of sensory nerve myelination during aging

Jha, M. K., Lee, Y., Russell, K. A., Yang, F., Dastgheyb, R. M., Deme, P., Ament, X. H., Chen, W., Liu, Y., Guan, Y., Polydefkis, M. J., Hoke, A., Haughey, N. J., Rothstein, J. D. & Morrison, B. M., Jan 1 2020, In: Glia. 68, 1, p. 161-177 17 p.

Research output: Contribution to journal › Article › peer-review

Open Access

15 Scopus citations

Optimizing Nervous System-Specific Gene Targeting with Cre Driver Lines: Prevalence of Germline Recombination and Influencing Factors

Luo, L., Ambrozkiewicz, M. C., Benseler, F., Chen, C., Dumontier, E., Falkner, S., Furlanis, E., Gomez, A. M., Hoshina, N., Huang, W. H., Hutchison, M. A., Itoh-Maruoka, Y., Lavery, L. A., Li, W., Maruo, T., Motohashi, J., Pai, E. L. L., Pelkey, K. A., Pereira, A. & Philips, T. & 36 others, Sinclair, J. L., Stogsdill, J. A., Traunmüller, L., Wang, J., Wortel, J., You, W., Abumaria, N., Beier, K. T., Brose, N., Burgess, H. A., Cepko, C. L., Cloutier, J. F., Eroglu, C., Goebbels, S., Kaeser, P. S., Kay, J. N., Lu, W., Luo, L., Mandai, K., McBain, C. J., Nave, K. A., Prado, M. A. M., Prado, V. F., Rothstein, J., Rubenstein, J. L. R., Saher, G., Sakimura, K., Sanes, J. R., Scheiffele, P., Takai, Y., Umemori, H., Verhage, M., Yuzaki, M., Zoghbi, H. Y., Kawabe, H. & Craig, A. M., Apr 8 2020, In: Neuron. 106, 1, p. 37-65.e5

Research output: Contribution to journal › Article › peer-review

Open Access

22 Scopus citations

Tfeb/mitf links impaired nuclear import to autophagolysosomal dysfunction in c9-als

Cunningham, K. M., Maulding, K., Ruan, K., Senturk, M., Grima, J. C., Sung, H., Zuo, Z., Song, H., Gao, J., Dubey, S., Rothstein, J. D., Zhang, K., Bellen, H. J. & Lloyd, T. E., Dec 2020, In: eLife. 9, p. 1-35 35 p., e59419.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Trial of sodium phenylbutyrate-taurursodiol for amyotrophic lateral sclerosis

Paganoni, S., Macklin, E. A., Hendrix, S., Berry, J. D., Elliott, M. A., Maiser, S., Karam, C., Caress, J. B., Owegi, M. A., Quick, A., Wymer, J., Goutman, S. A., Heitzman, D., Heiman-Patterson, T., Jackson, C. E., Quinn, C., Rothstein, J. D., Kasarskis, E. J., Katz, J. & Jenkins, L. & 39 others, Ladha, S., Miller, T. M., Scelsa, S. N., Vu, T. H., Fournier, C. N., Glass, J. D., Johnson, K. M., Swenson, A., Goyal, N. A., Pattee, G. L., Andres, P. L., Babu, S., Chase, M., Dagostino, D., Dickson, S. P., Ellison, N., Hall, M., Hendrix, K., Kittle, G., McGovern, M., Ostrow, J., Pothier, L., Randall, R., Shefner, J. M., Sherman, A. V., Tustison, E., Vigneswaran, P., Walker, J., Yu, H., Chan, J., Wittes, J., Cohen, J., Klee, J., Leslie, K., Tanzi, R. E., Gilbert, W., Yeramian, P. D., Schoenfeld, D. & Cudkowicz, M. E., Sep 3 2020, In: New England Journal of Medicine. 383, 10, p. 919-930 12 p.

Research output: Contribution to journal › Article › peer-review

Open Access

21 Scopus citations

jeff Rothstein

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