• 12479
1992 …2022

Research activity per year

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Personal profile

Research interests

My research program focuses on:

1. Gene down regulation strategies (RNAi and antisense oligonucleotides) to understand disease pathogenesis and provide novel therapies for neurodegenerative diseases, in particular amyotrophic lateral sclerosis (ALS)

2. How dysfunction of mitochondria causes loss of motor neurons.

Clinical interests

ALS and neurological disorders


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  • Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

    for the Healey ALS Platform Trial Study Group, HEALEY ALS Platform Trial Study Group, Biostatisticians, Investigators, Clinical Operations Team & Medical Monitors, Feb 2022, In: Annals of neurology. 91, 2, p. 165-175 11 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    8 Scopus citations
  • 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 othersPerez, 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 journalArticlepeer-review

    Open Access
    11 Scopus citations
  • Antisense Oligonucleotides for the Study and Treatment of ALS

    Boros, B. D., Schoch, K. M., Kreple, C. J. & Miller, T., Jul 2022, In: Neurotherapeutics. 19, 4, p. 1145-1158 14 p.

    Research output: Contribution to journalReview articlepeer-review

    3 Scopus citations
  • Astrocytic 4R tau expression drives astrocyte reactivity and dysfunction

    Ezerskiy, L. A., Schoch, K. M., Sato, C., Beltcheva, M., Horie, K., Rigo, F., Martynowicz, R., Karch, C. M., Bateman, R. J. & Miller, T. M., Jan 11 2022, In: JCI Insight. 7, 1, 152012.

    Research output: Contribution to journalArticlepeer-review

    Open Access
  • Cell environment shapes TDP-43 function with implications in neuronal and muscle disease

    NYGC ALS Consortium, Dec 2022, In: Communications Biology. 5, 1, 314.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    2 Scopus citations