Rigo, Frank

Robust transcriptome-wide discovery of RNA-binding protein binding sites with enhanced CLIP (eCLIP)

Van Nostrand, E. L., Pratt, G. A., Shishkin, A. A., Gelboin-Burkhart, C., Fang, M. Y., Sundararaman, B., Blue, S. M., Nguyen, T. B., Surka, C., Elkins, K., et al. (2016) · Nature Methods

RNA Research and SplicingGenomics and Chromatin Dynamics

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

Donnelly, C. J., Zhang, P.-W., Pham, J. T., Haeusler, A. R., Mistry, N. A., Vidensky, S., Daley, E. L., Poth, E. M., Hoover, B., Fines, D. M., et al. (2013) · Neuron

Amyotrophic Lateral Sclerosis ResearchGenetic Neurodegenerative Diseases

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Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion

Sareen, D., O’Rourke, J. G., Meera, P., Muhammad, A. K. M. G., Grant, S., Simpkinson, M., Bell, S., Carmona, S., Ornelas, L., Sahabian, A., et al. (2013) · Science Translational Medicine

Amyotrophic Lateral Sclerosis ResearchGenetic Neurodegenerative Diseases

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Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration

Melamed, Z., López-Erauskin, J., Baughn, M. W., Zhang, O., Drenner, K., Sun, Y., Freyermuth, F., McMahon, M. A., Beccari, M. S., Artates, J. W., et al. (2019) · Nature Neuroscience

Amyotrophic Lateral Sclerosis ResearchRNA Research and Splicing

Cited by 635

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

Becker, L. A., Huang, B., Bieri, G., Ma, R., Knowles, D. A., Jafar-Nejad, P., Messing, J., Kim, H. J., Soriano, A., Auburger, G., et al. (2017) · Nature

Amyotrophic Lateral Sclerosis ResearchGenetic Neurodegenerative Diseases

Cited by 571

Gain of toxicity from ALS/FTD-linked repeat expansions in C9ORF72 is alleviated by antisense oligonucleotides targeting GGGGCC-containing RNAs

Jiang, J., Zhu, Q., Gendron, T. F., Saberi, S., McAlonis-Downes, M., Seelman, A., Stauffer, J. E., Jafar-nejad, P., Drenner, K., Schulte, D., et al. (2016) · Neuron

Amyotrophic Lateral Sclerosis ResearchGenetic Neurodegenerative Diseases

Cited by 536

The long non-coding RNA Gomafu is acutely regulated in response to neuronal activation and involved in schizophrenia-associated alternative splicing

Barry, G., Briggs, J. A., Vanichkina, D. P., Poth, E. M., Beveridge, N. J., Ratnu, V. S., Nayler, S. P., Nones, K., Hu, J., Bredy, T. W., et al. (2014) · Molecular Psychiatry

Cancer-related molecular mechanisms researchRNA Research and Splicing

Cited by 394

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

Scoles, D. R., Meera, P., Schneider, M. D., Paul, S., Dansithong, W., Figueroa, K. P., Hung, G., Rigo, F., Bennett, C. F., Otis, T. S., et al. (2017) · Nature

Genetic Neurodegenerative DiseasesAmyotrophic Lateral Sclerosis Research

Cited by 319

Antisense Reduction of Tau in Adult Mice Protects against Seizures

DeVos, S. L., Goncharoff, D. K., Chen, G., Kebodeaux, C. S., Yamada, K., Stewart, F. R., Schuler, D. R., Maloney, S. E., Wozniak, D. F., Rigo, F., et al. (2013) · Journal of Neuroscience

Epilepsy research and treatmentAlzheimer's disease research and treatments

Cited by 311

Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

Rigo, F., Chun, S. J., Norris, D. A., Hung, G., Lee, S., Matson, J., Fey, R. A., Gaus, H., Hua, Y., Grundy, J. S., et al. (2014) · The Journal of Pharmacology and Experimental Therapeutics

Neurogenetic and Muscular Disorders ResearchRNA Interference and Gene Delivery

Cited by 283

Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72 -associated amyotrophic lateral sclerosis

Gendron, T. F., Chew, J., Stankowski, J. N., Hayes, L. R., Zhang, Y.-J., Prudencio, M., Carlomagno, Y., Daughrity, L. M., Jansen-West, K., Perkerson, E. A., et al. (2017) · Science Translational Medicine

Amyotrophic Lateral Sclerosis ResearchAlzheimer's disease research and treatments

Cited by 265

C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy

Cook, C. N., Wu, Y., Odeh, H. M., Gendron, T. F., Jansen-West, K., del Rosso, G., Yue, M., Jiang, P., Gomes, E., Tong, J., et al. (2020) · Science Translational Medicine

Amyotrophic Lateral Sclerosis ResearchGenetic Neurodegenerative Diseases

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Increased 4R-Tau Induces Pathological Changes in a Human-Tau Mouse Model

Schoch, K. M., DeVos, S. L., Miller, R. L., Chun, S. J., Norrbom, M., Wozniak, D. F., Dawson, H. N., Bennett, C. F., Rigo, F., & Miller, T. M. (2016) · Neuron

Alzheimer's disease research and treatmentsAmyotrophic Lateral Sclerosis Research

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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., et al. (2020) · Neuron

Amyotrophic Lateral Sclerosis ResearchNuclear Structure and Function

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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., Lusk, C. P., & Rothstein, J. D. (2021) · Science Translational Medicine

Amyotrophic Lateral Sclerosis ResearchNuclear Structure and Function

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Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier

Barker, S. J., Thayer, M. B., Kim, C., Tatarakis, D., Simon, M. J., Dial, R., Nilewski, L., Wells, R. C., Zhou, Y., Afetian, M., et al. (2024) · Science Translational Medicine

RNA Interference and Gene DeliveryBarrier Structure and Function Studies

Cited by 142

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Jafar-nejad, P., Powers, B., Soriano, A., Zhao, H., Norris, D. A., Matson, J., DeBrosse-Serra, B., Watson, J., Narayanan, P., Chun, S. J., et al. (2021) · Nucleic Acids Research

RNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

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Therapeutic correction of ApoER2 splicing in Alzheimer's disease mice using antisense oligonucleotides

Hinrich, A. J., Jodelka, F. M., Chang, J. L., Brutman, D., Bruno, A. M., Briggs, C. A., James, B. D., Stutzmann, G. E., Bennett, D. A., Miller, S. A., et al. (2016) · EMBO Molecular Medicine

Alzheimer's disease research and treatmentsRNA Research and Splicing

Cited by 127

Antisense oligonucleotide therapy for KCNT1 encephalopathy

Burbano, L. E., Li, M., Jancovski, N., Jafar-Nejad, P., Richards, K., Sedo, A., Soriano, A., Rollo, B., Jia, L., Gazina, E. V., et al. (2022) · JCI Insight

Epilepsy research and treatmentIon channel regulation and function

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Acute Trem2 reduction triggers increased microglial phagocytosis, slowing amyloid deposition in mice

Schoch, K. M., Ezerskiy, L. A., Morhaus, M. M., Bannon, R. N., Sauerbeck, A. D., Shabsovich, M., Jafar-nejad, P., Rigo, F., & Miller, T. M. (2021) · Proceedings of the National Academy of Sciences

Neuroinflammation and Neurodegeneration MechanismsAlzheimer's disease research and treatments

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Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB

Maimon, R., Chillon-Marinas, C., Snethlage, C. E., Singhal, S. M., McAlonis-Downes, M., Ling, K., Rigo, F., Bennett, C. F., Da Cruz, S., Hnasko, T. S., et al. (2021) · Nature Neuroscience

Neurogenesis and neuroplasticity mechanismsNeuroscience and Neuropharmacology Research

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Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2

Hill, S. F., Ziobro, J. M., Jafar‐Nejad, P., Rigo, F., & Meisler, M. H. (2022) · Epilepsia

Ion channel regulation and functionEpilepsy research and treatment

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Reduction of nemo-like kinase increases lysosome biogenesis and ameliorates TDP-43–related neurodegeneration

Tejwani, L., Jung, Y., Kokubu, H., Sowmithra, S., Ni, L., Lee, C., Sanders, B., Lee, P. J., Xiang, Y., Luttik, K., et al. (2023) · Journal of Clinical Investigation

Amyotrophic Lateral Sclerosis ResearchAutophagy in Disease and Therapy

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