Nicholas J Maragakis

Professor

1992 …2020
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Research Output 1992 2020

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2020

Focal and dose-dependent neuroprotection in ALS mice following AAV2-neurturin delivery

Gross, S. K., Shim, B. S., Bartus, R. T., Deaver, D., McEachin, Z., Bétourné, A., Boulis, N. M. & Maragakis, N. J., Jan 2020, In : Experimental Neurology. 323, 113091.

Research output: Contribution to journalArticle

Neurturin
Dependovirus
Neuromuscular Junction
Nerve Growth Factors
Motor Neurons
2019

Perfluorocarbon Labeling of Human Glial-Restricted Progenitors for 19F Magnetic Resonance Imaging

Richard, J-P., Hussain, U., Gross, S., Taga, A., Kouser, M., Almad, A., Campanelli, J. T., Bulte, J. W. & Maragakis, N. J., Jan 1 2019, (Accepted/In press) In : Stem cells translational medicine.

Research output: Contribution to journalArticle

Fluorocarbons
Neuroglia
Magnetic Resonance Imaging
Amyotrophic Lateral Sclerosis
Transplantation

Role of Human-Induced Pluripotent Stem Cell-Derived Spinal Cord Astrocytes in the Functional Maturation of Motor Neurons in a Multielectrode Array System

Taga, A., Dastgheyb, R., Habela, C., Joseph, J., Richard, J. P., Gross, S. K., Lauria, G., Lee, G., Haughey, N. & Maragakis, N. J., Jan 1 2019, (Accepted/In press) In : Stem Cells Translational Medicine.

Research output: Contribution to journalArticle

Open Access
Induced Pluripotent Stem Cells
Motor Neurons
Astrocytes
Spinal Cord
Coculture Techniques

Shared polygenic risk and causal inferences in amyotrophic lateral sclerosis

The ITALSGEN Consortium & The International ALS Genomics Consortium, Apr 1 2019, In : Annals of neurology. 85, 4, p. 470-481 12 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Genome-Wide Association Study
Linkage Disequilibrium
Random Allocation
Smoking
2018

ALSUntangled 41: “Eric Is Winning”

THE ALSUNTANGLED GROUP, Jan 2 2018, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 19, 1-2, p. 157-160 4 p.

Research output: Contribution to journalArticle

ALSUntangled 43: copper

THE ALSUNTANGLED GROUP, Jul 3 2018, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 19, 5-6, p. 472-476 5 p.

Research output: Contribution to journalArticle

Copper

A stocked toolbox for understanding the role of astrocytes in disease

Almad, A. & Maragakis, N. J., May 16 2018, (Accepted/In press) In : Nature Reviews Neurology. p. 1-12 12 p.

Research output: Contribution to journalArticle

Astrocytes
Nervous System Diseases
Optogenetics
Induced Pluripotent Stem Cells
Disease Progression

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene

ITALSGEN Consortium, Genomic Translation for ALS Care (GTAC) Consortium, ALS Sequencing Consortium, NYGC ALS Consortium, Answer ALS Foundation, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, SLAGEN Consortium, French ALS Consortium & Project MinE ALS Sequencing Consortium, Mar 21 2018, In : Neuron. 97, 6, p. 1268-1283.e6

Research output: Contribution to journalArticle

Kinesin
Genome
Mutation
Genes
Genome-Wide Association Study

Motor neuron-derived microRNAs cause astrocyte dysfunction in amyotrophic lateral sclerosis

Hoye, M. L., Regan, M. R., Jensen, L. A., Lake, A. M., Reddy, L. V., Vidensky, S., Richard, J-P., Maragakis, N. J., Rothstein, J. D., Dougherty, J. D. & Miller, T. M., Sep 1 2018, In : Brain : a journal of neurology. 141, 9, p. 2561-2575 15 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Motor Neurons
MicroRNAs
Astrocytes
Excitatory Amino Acid Transporter 2

Olfactory dysfunction in amyotrophic lateral sclerosis

Viguera, C., Wang, J., Mosmiller, E., Cerezo, A. & Maragakis, N. J., Aug 1 2018, In : Annals of Clinical and Translational Neurology. 5, 8, p. 976-981 6 p.

Research output: Contribution to journalArticle

Smell
Amyotrophic Lateral Sclerosis
Linear Models
Olfaction Disorders
Phenotype
2017

ALSUntangled 38: L-serine

THE ALSUNTANGLED GROUP, Jan 2 2017, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 18, 1-2, p. 148-151 4 p.

Research output: Contribution to journalArticle

ALSUntangled No. 36: Accilion

THE ALSUNTANGLED GROUP, Jan 2 2017, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 18, 1-2, p. 142-147 6 p.

Research output: Contribution to journalArticle

ALSUntangled No. 37: Inosine*

THE ALSUNTANGLED GROUP, Apr 3 2017, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 18, 3-4, p. 309-312 4 p.

Research output: Contribution to journalArticle

A randomized controlled trial of resistance and endurance exercise in amyotrophic lateral sclerosis

On behalf of the neals consortium, Nov 29 2017, (Accepted/In press) In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. p. 1-9 9 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Randomized Controlled Trials
Exercise
Outcome Assessment (Health Care)
Physical Therapists

Fibroblast bioenergetics to classify amyotrophic lateral sclerosis patients

Konrad, C., Kawamata, H., Bredvik, K. G., Arreguin, A. J., Cajamarca, S. A., Hupf, J. C., Ravits, J. M., Miller, T. M., Maragakis, N. J., Hales, C. M., Glass, J. D., Gross, S., Mitsumoto, H. & Manfredi, G., Oct 24 2017, In : Molecular Neurodegeneration. 12, 1, 1 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Energy Metabolism
Fibroblasts
Glycolysis
Respiration

MicroRNA profiling reveals marker of motor neuron disease in ALS models

Hoye, M. L., Koval, E. D., Wegener, A. J., Hyman, T. S., Yang, C., O’Brien, D. R., Miller, R. L., Cole, T., Schoch, K. M., Shen, T., Kunikata, T., Richard, J-P., Gutmann, D. H., Maragakis, N. J., Kordasiewicz, H. B., Dougherty, J. D. & Miller, T. M., May 31 2017, In : Journal of Neuroscience. 37, 22, p. 5574-5586 13 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Motor Neurons
MicroRNAs
RNA
Rodentia

Serial in vivo imaging of transplanted allogeneic neural stem cell survival in a mouse model of amyotrophic lateral sclerosis

Srivastava, A. K., Gross, S. K., Almad, A., Bulte, C. A., Maragakis, N. J. & Bulte, J. W., Mar 1 2017, In : Experimental Neurology. 289, p. 96-102 7 p.

Research output: Contribution to journalArticle

Neural Stem Cells
Amyotrophic Lateral Sclerosis
Cell Survival
Transplantation
Rotarod Performance Test

Sporadic ALS Astrocytes Induce Neuronal Degeneration In Vivo

Qian, K., Huang, H., Peterson, A., Hu, B., Maragakis, N. J., Ming, G. L., Chen, H. & Zhang, S. C., Apr 11 2017, In : Stem Cell Reports. 8, 4, p. 843-855 13 p.

Research output: Contribution to journalArticle

Motor Neurons
Astrocytes
Neurons
Induced Pluripotent Stem Cells
Motor Neuron Disease
2016

ALSUntangled No. 34: GM604

THE ALSUNTANGLED GROUP, Nov 16 2016, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 17, 7-8, p. 617-621 5 p.

Research output: Contribution to journalArticle

ALSUntangled No. 35: Hyperbaric Oxygen Therapy*

THE ALSUNTANGLED GROUP, Nov 16 2016, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 17, 7-8, p. 622-624 3 p.

Research output: Contribution to journalArticle

A Spontaneous Missense Mutation in Branched Chain Keto Acid Dehydrogenase Kinase in the Rat Affects Both the Central and Peripheral Nervous Systems

Zigler, J. S., Hodgkinson, C. A., Wright, M., Klise, A., Sundin, O., Broman, K. W., Hejtmancik, F., Huang, H., Patek, B., Sergeev, Y., Hose, S., Brayton, C., Xaiodong, J., Vasquez, D., Maragakis, N., Mori, S., Goldman, D., Hoke, A. & Sinha, D., 2016, In : PLoS One. 11, 7, p. e0160447

Research output: Contribution to journalArticle

3-methyl-2-oxobutanoate dehydrogenase (lipoamide)
3-Methyl-2-Oxobutanoate Dehydrogenase (Lipoamide)
Branched Chain Amino Acids
peripheral nervous system
missense mutation

Astrocytes drive upregulation of the multidrug resistance transporter ABCB1 (P-Glycoprotein) in endothelial cells of the blood–brain barrier in mutant superoxide dismutase 1-linked amyotrophic lateral sclerosis

Qosa, H., Lichter, J., Sarlo, M., Markandaiah, S. S., McAvoy, K., Richard, J-P., Jablonski, M. R., Maragakis, N. J., Pasinelli, P. & Trotti, D., Aug 1 2016, In : GLIA. 64, 8, p. 1298-1313 16 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Multiple Drug Resistance
P-Glycoprotein
Astrocytes
Up-Regulation

Connexin 43 in astrocytes contributes to motor neuron toxicity in amyotrophic lateral sclerosis

Almad, A., Doreswamy, A., Gross, S. K., Richard, J-P., Huo, Y., Haughey, N. & Maragakis, N. J., 2016, (Accepted/In press) In : GLIA.

Research output: Contribution to journalArticle

Connexin 43
Amyotrophic Lateral Sclerosis
Motor Neurons
Astrocytes
Connexins

Defining SOD1 ALS natural history to guide therapeutic clinical trial design

Bali, T., Self, W., Liu, J., Siddique, T., Wang, L. H., Bird, T. D., Ratti, E., Atassi, N., Boylan, K. B., Glass, J. D., Maragakis, N. J., Caress, J. B., McCluskey, L. F., Appel, S. H., Wymer, J. P., Gibson, S., Zinman, L., Mozaffar, T., Callaghan, B., McVey, A. L. & 7 others, Jockel-Balsarotti, J., Allred, P., Fisher, E. R., Lopate, G., Pestronk, A., Cudkowicz, M. E. & Miller, T. M., Jun 3 2016, (Accepted/In press) In : Journal of Neurology, Neurosurgery and Psychiatry.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Natural History
Clinical Trials
Mutation
Survival

Generation of GFAP::GFP astrocyte reporter lines from human adult fibroblast-derived iPS cells using zinc-finger nuclease technology

Zhang, P., Haidet-Phillips, A. M., Pham, J. T., Lee, Y., Huo, Y., Tienari, P. J., Maragakis, N. J., Sattler, R. & Rothstein, J. D., Jan 1 2016, In : GLIA. 64, 1, p. 63-75 13 p.

Research output: Contribution to journalArticle

Glial Fibrillary Acidic Protein
Zinc Fingers
Astrocytes
Fibroblasts
Technology

Primary lateral sclerosis and early upper motor neuron disease: Characteristics of a cross-sectional population

Fournier, C. N., Murphy, A., Loci, L., Mitsumoto, H., Lomen-Hoerth, C., Kisanuki, Y., Simmons, Z., Maragakis, N. J., McVey, A. L., Al-Lahham, T., Heiman-Patterson, T. D., Andrews, J., McDonnell, E., Cudkowicz, M. & Atassi, N., 2016, In : Journal of Clinical Neuromuscular Disease. 17, 3, p. 99-105 7 p.

Research output: Contribution to journalArticle

Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Electromyography
Population
Hereditary Spastic Paraplegia
2015

A comprehensive library of familial human amyotrophic lateral sclerosis induced pluripotent stem cells

Li, Y., Balasubramanian, U., Cohen, D., Zhang, P., Mosmiller, E., Sattler, R., Maragakis, N. J. & Rothstein, J. D., Mar 11 2015, In : PLoS One. 10, 3, e0118266.

Research output: Contribution to journalArticle

Induced Pluripotent Stem Cells
Stem cells
Libraries
mutation
Mutation

ALSUntangled: Introducing the table of evidence

ALSUntangled Group, Mar 1 2015, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 16, 1-2, p. 142-145 4 p.

Research output: Contribution to journalArticle

ALSUntangled No. 29: MitoQ

The Alsuntangled Lsuntangled Grouproup, Aug 27 2015, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 16, 5-6, p. 427-429 3 p.

Research output: Contribution to journalArticle

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

Choi, I. Y., Lim, H. T., Estrellas, K., Mula, J., Cohen, T. V., Zhang, Y., Donnelly, C. J., Richard, J-P., Kim, Y. J., Kim, H., Kazuki, Y., Oshimura, M., Li, H. L., Hotta, A., Rothstein, J. D., Maragakis, N. J., Wagner, K. R. & Lee, G., Sep 9 2015, (Accepted/In press) In : Cell Reports.

Research output: Contribution to journalArticle

Duchenne Muscular Dystrophy
Myoblasts
Phenotype
Chemical compounds
Stem cells

Hemiparetic primary lateral sclerosis: Revisiting mills syndrome

Maragakis, N. J., Holland, N. R. & Corse, A. M., 2015, In : Case Reports in Neurology. 7, 3, p. 191-195 5 p.

Research output: Contribution to journalArticle

Motor Neuron Disease
Licensure
Pharmaceutical Preparations
Publications
Extremities

Human glial progenitor engraftment and gene expression is independent of the ALS environment

Haidet-Phillips, A. M., Doreswamy, A., Gross, S. K., Tang, X., Campanelli, J. T. & Maragakis, N. J., Feb 1 2015, In : Experimental Neurology. 264, p. 188-199 12 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Neuroglia
Astrocytes
Spinal Cord
Gene Expression

Human iPS cell-derived astrocyte transplants preserve respiratory function after spinal cord injury

Li, K., Javed, E., Scura, D., Hala, T. J., Seetharam, S., Falnikar, A., Richard, J-P., Chorath, A., Maragakis, N. J., Wright, M. C. & Lepore, A. C., 2015, In : Experimental Neurology. 271, p. 479-492 14 p.

Research output: Contribution to journalArticle

Induced Pluripotent Stem Cells
Spinal Cord Injuries
Astrocytes
Transplants
Transplantation

Induced pluripotent stem cells from ALS patients for disease modeling

Richard, J-P. & Maragakis, N. J., May 14 2015, In : Brain Research. 1607, p. 15-25 11 p.

Research output: Contribution to journalArticle

Induced Pluripotent Stem Cells
Amyotrophic Lateral Sclerosis
Motor Neurons
Germ Layers
Pluripotent Stem Cells

Neural and glial progenitor transplantation as a neuroprotective strategy for Amyotrophic Lateral Sclerosis (ALS)

Haidet-Phillips, A. M. & Maragakis, N. J., Dec 2 2015, In : Brain Research. 1628, p. 343-350 8 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Neuroglia
Transplantation
Stem Cell Transplantation
Stem Cells

Peripheral neuropathy in a family with Sandhoff disease and SH3TC2 deficiency

Grunseich, C., Schindler, A. B., Chen, K. L., Bakar, D., Mankodi, A., Traslavina, R., Ray-Chaudhury, A., Lehky, T. J., Baker, E. H., Maragakis, N. J., Tifft, C. J. & Fischbeck, K. H., Apr 1 2015, In : Journal of Neurology. 262, 4, p. 1066-1068 3 p.

Research output: Contribution to journalArticle

beta-Hexosaminidase beta Chain
Sandhoff Disease
Peripheral Nervous System Diseases
Pedigree
Proteins

Pre-morbid type 2 diabetes mellitus is not a prognostic factor in amyotrophic lateral sclerosis

Paganoni, S., Hyman, T., Shui, A., Allred, P., Harms, M., Liu, J., Maragakis, N. J., Schoenfeld, D., Yu, H., Atassi, N., Cudkowicz, M. & Miller, T. M., Sep 1 2015, In : Muscle and Nerve. 52, 3, p. 339-343 5 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Type 2 Diabetes Mellitus
Clinical Trials
Body Mass Index
Multivariate Analysis

Transplantation of glial progenitors that overexpress glutamate transporter GLT1 preserves diaphragm function following cervical SCI

Li, K., Javed, E., Hala, T. J., Sannie, D., Regan, K. A., Maragakis, N. J., Wright, M. C., Poulsen, D. J. & Lepore, A. C., Mar 5 2015, In : Molecular Therapy. 23, 3, p. 533-548 16 p.

Research output: Contribution to journalArticle

Amino Acid Transport System X-AG
Diaphragm
Spinal Cord Injuries
Neuroglia
Transplantation
2014

ALSUntangled no. 26: Lunasin

ALSUntangled Group, Dec 1 2014, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 15, 7-8, p. 622-626 5 p.

Research output: Contribution to journalArticle

C9orf72 nucleotide repeat structures initiate molecular cascades of disease

Haeusler, A. R., Donnelly, C. J., Periz, G., Simko, E. A. J., Shaw, P. G., Kim, M. S., Maragakis, N. J., Troncoso, J. C., Pandey, A., Sattler, R., Rothstein, J. D. & Wang, J., 2014, In : Nature. 507, 7491, p. 195-200 6 p.

Research output: Contribution to journalArticle

G-Quadruplexes
Molecular Structure
Nucleotides
Neurodegenerative Diseases
Pathology

Electrical impedance myography correlates with standard measures of ALS severity

Rutkove, S. B., Caress, J. B., Cartwright, M. S., Burns, T. M., Warder, J., David, W. S., Goyal, N., Maragakis, N. J., Benatar, M., Sharma, K. R., Narayanaswami, P., Raynor, E. M., Watson, M. L. & Shefner, J. M., 2014, In : Muscle and Nerve. 49, 3, p. 441-443 3 p.

Research output: Contribution to journalArticle

Myography
Amyotrophic Lateral Sclerosis
Electric Impedance
Lower Extremity
Natural History

Gene Profiling of Human Induced Pluripotent Stem Cell-Derived Astrocyte Progenitors Following Spinal Cord Engraftment

Haidet-Phillips, A. M., Roybon, L., Gross, S. K., Tuteja, A., Donnelly, C. J., Richard, J-P., Ko, M., Sherman, A., Eggan, K., Henderson, C. E. & Maragakis, N. J., 2014, In : Stem cells translational medicine. 3, 5, p. 575-585 11 p.

Research output: Contribution to journalArticle

Induced Pluripotent Stem Cells
Astrocytes
Spinal Cord
Genes
Stem Cell Transplantation
2013

ALS untangled no. 20: The Deanna protocol

Fournier, C., Bedlack, R., Hardiman, O., Heiman-Patterson, T., Gutmann, L., Bromberg, M., Ostrow, L., Carter, G., Kabashi, E., Bertorini, T., Mozaffar, T., Andersen, P., Dietz, J., Gamez, J., Dimachkie, M., Wang, Y., Wicks, P., Heywood, J., Novella, S., Rowland, L. P. & 69 others, Pioro, E., Kinsley, L., Mitchell, K., Glass, J., Sathornsumetee, S., Kwiecinski, H., Baker, J., Atassi, N., Forshew, D., Ravits, J., Conwit, R., Jackson, C., Sherman, A., Dalton, K., Tindall, K., Gonzalez, G., Robertson, J., Phillips, L., Benatar, M., Sorenson, E., Shoesmith, C., Nash, S., Maragakis, N. J., Moore, D., Caress, J., Boylan, K., Armon, C., Grosso, M., Gerecke, B., Wymer, J., Oskarsson, B., Bowser, R., Drory, V., Shefner, J., Lechtzin, N., Leitner, M., Miller, R., Mitsumoto, H., Levine, T., Russell, J., Sharma, K., Saperstein, D., McClusky, L., MacGowan, D., Licht, J., Verma, A., Strong, M., Lomen-Hoerth, C., Tandan, R., Rivner, M., Kolb, S., Polak, M., Rudnicki, S., Kittrell, P., Quereshi, M., Sachs, G., Pattee, G., Weiss, M., Kissel, J., Goldstein, J., Rothstein, J. D., Pastula, D., Levitsky, G., Ogino, M., Rosenfeld, J., Carmi, E., Oster, C., Barkhaus, P. & Valor, E., May 2013, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 14, 4, p. 319-323 5 p.

Research output: Contribution to journalArticle

Altered astrocytic expression of TDP-43 does not influence motor neuron survival

Haidet-Phillips, A. M., Gross, S. K., Williams, T., Tuteja, A., Sherman, A., Ko, M., Jeong, Y. H., Wong, P. C. & Maragakis, N. J., Dec 2013, In : Experimental Neurology. 250, p. 250-259 10 p.

Research output: Contribution to journalArticle

Tars
DNA-Binding Proteins
Motor Neurons
Astrocytes
Amyotrophic Lateral Sclerosis

Exome sequencing to identify de novo mutations in sporadic ALS trios

Chesi, A., Staahl, B. T., Jovičić, A., Couthouis, J., Fasolino, M., Raphael, A. R., Yamazaki, T., Elias, L., Polak, M., Kelly, C., Williams, K. L., Fifita, J. A., Maragakis, N. J., Nicholson, G. A., King, O. D., Reed, R., Crabtree, G. R., Blair, I. P., Glass, J. D. & Gitler, A. D., 2013, In : Nature Neuroscience. 16, 7, p. 851-855 5 p.

Research output: Contribution to journalArticle

Exome
Amyotrophic Lateral Sclerosis
Mutation
RNA-Binding Protein FUS
Neuronal Plasticity

Mechanisms, models and biomarkers in amyotrophic lateral sclerosis

Turner, M. R., Bowser, R., Bruijn, L., Dupuis, L., Ludolph, A., McGrath, M., Manfredi, G., Maragakis, N. J., Miller, R. G., Pullman, S. L., Rutkove, S. B., Shaw, P. J., Shefner, J. & Fischbeck, K. H., May 2013, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 14, SUPPL1, p. 19-32 14 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Motor Neurons
Biomarkers
Induced Pluripotent Stem Cells
Frontotemporal Dementia

Optical coherence tomography does not support optic nerve involvement in amyotrophic lateral sclerosis

Roth, N. M., Saidha, S., Zimmermann, H., Brandt, A. U., Oberwahrenbrock, T., Maragakis, N. J., Tumani, H., Ludolph, A. C., Meyer, T., Calabresi, P. & Paul, F., Aug 2013, In : European Journal of Neurology. 20, 8, p. 1170-1176 7 p.

Research output: Contribution to journalArticle

Optical Coherence Tomography
Amyotrophic Lateral Sclerosis
Optic Nerve
Visual Pathways
Nerve Fibers

Peer recommendations on how to improve clinical research, and Conference wrap-up

Chad, D. A., Rowland, L. P., Armon, C., Bedlack, R., Durham, H., Factor-Litvak, P., Heiman-Patterson, T., Heitzman, D., Lacomis, D., Ludolph, A., Maragakis, N. J., Miller, R., Pattee, G., Shoesmith, C., Sorenson, E. & Turner, M. R., May 2013, In : Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 14, SUPPL1, p. 67-73 7 p.

Research output: Contribution to journalArticle

Research
Epidemiologic Studies
Clinical Trials
Physicians

RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention

Donnelly, C. J., Zhang, P., Pham, J. T., Heusler, A. R., Mistry, N. A., Vidensky, S., Daley, E. L., Poth, E. M., Hoover, B., Fines, D. M., Maragakis, N. J., Tienari, P. J., Petrucelli, L., Traynor, B. J., Wang, J., Rigo, F., Bennett, C. F., Blackshaw, S., Sattler, R. & Rothstein, J. D., Oct 16 2013, In : Neuron. 80, 2, p. 415-428 14 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
RNA
Induced Pluripotent Stem Cells
Frontotemporal Dementia
RNA-Binding Proteins

RNA Toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention [Neuron, 79, (September 18, 2013), 1222-1231]

Donnelly, C. J., Zhang, P., Pham, J. T., Haeusler, A. R., Mistry, N. A., Vidensky, S., Daley, E. L., Poth, E. M., Hoover, B., Fines, D. M., Maragakis, N. J., Tienari, P. J., Petrucelli, L., Traynor, B. J., Wang, J., Rigo, F., Bennett, C. F., Blackshaw, S., Sattler, R. & Rothstein, J. D., Nov 20 2013, In : Neuron. 80, 4, p. 1102 1 p.

Research output: Contribution to journalArticle

RNA
Neurons