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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Nature medicine, 2022-01, Vol.28 (1), p.104-116

2022

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Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome

Annals of neurology, 2020-03, Vol.87 (3), p.339-346

2020

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Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2

Epilepsia (Copenhagen), 2022-10, Vol.63 (10), p.e125-e131

2022

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A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis

Nature neuroscience, 2020-03, Vol.23 (3), p.386-397

2020

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TRIM28 regulates the nuclear accumulation and toxicity of both alpha-synuclein and tau

eLife, 2016-10, Vol.5

2016

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Correction: Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

eLife, 2024-10, Vol.13

2024

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Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

eLife, 2023-01, Vol.12

2023

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

Nature (London), 2017-04, Vol.544 (7650), p.367-371

2017

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Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

Neuron (Cambridge, Mass.), 2016-05, Vol.90 (3), p.535-550

2016

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

Nucleic acids research, 2021-01, Vol.49 (2), p.657-673

2021

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A native interactor scaffolds and stabilizes toxic ATAXIN-1 oligomers in SCA1

eLife, 2015-05, Vol.4

2015

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Therapeutic inhibition of mTORC2 rescues the behavioral and neurophysiological abnormalities associated with Pten-deficiency

Nature medicine, 2019-11, Vol.25 (11), p.1684-1690

2019

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Sarm1 haploinsufficiency or low expression levels after antisense oligonucleotides delay programmed axon degeneration

Cell reports (Cambridge), 2021-12, Vol.37 (11), p.110108-110108, Article 110108

2021

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Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy

Frontiers in neuroscience, 2023-10, Vol.17, p.1282201

2023

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Reduction of Nuak1 Decreases Tau and Reverses Phenotypes in a Tauopathy Mouse Model

Neuron (Cambridge, Mass.), 2016-10, Vol.92 (2), p.407-418

2016

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Targeted suppression of mTORC2 reduces seizures across models of epilepsy

Nature communications, 2023-11, Vol.14 (1), p.7364-7364, Article 7364

2023

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Pumilio1 Haploinsufficiency Leads to SCA1-like Neurodegeneration by Increasing Wild-Type Ataxin1 Levels

Cell, 2015-03, Vol.160 (6), p.1087-1098

2015

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Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1

Nature, 2008-04, Vol.452 (7188), p.713-718

2008

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Targeting nonsense-mediated RNA decay does not increase progranulin levels in the Grn R493X mouse model of frontotemporal dementia

PloS one, 2023-03, Vol.18 (3), p.e0282822

2023

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

The Journal of clinical investigation, 2023-08, Vol.133 (16), p.1-17

2023

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RAS–MAPK–MSK1 pathway modulates ataxin 1 protein levels and toxicity in SCA1

Nature (London), 2013-06, Vol.498 (7454), p.325-331

2013

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Differential effects of SARM1 inhibition in traumatic glaucoma and EAE optic neuropathies

Molecular therapy. Nucleic acids, 2023-06, Vol.32, p.13-27

2023

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PFTK1 kinase regulates axogenesis during development via RhoA activation

BMC biology, 2023-10, Vol.21 (1), p.1-240, Article 240

2023

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NMNAT2 supports vesicular glycolysis via NAD homeostasis to fuel fast axonal transport

Molecular neurodegeneration, 2024-01, Vol.19 (1), p.13-31, Article 13

2024

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Reduction of matrix metalloproteinase 9 (MMP-9) protects motor neurons from TDP-43-triggered death in rNLS8 mice

Neurobiology of disease, 2019-04, Vol.124, p.133-140

2019

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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome

Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2

A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis

TRIM28 regulates the nuclear accumulation and toxicity of both alpha-synuclein and tau

Correction: Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome

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

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

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

A native interactor scaffolds and stabilizes toxic ATAXIN-1 oligomers in SCA1

Therapeutic inhibition of mTORC2 rescues the behavioral and neurophysiological abnormalities associated with Pten-deficiency

Sarm1 haploinsufficiency or low expression levels after antisense oligonucleotides delay programmed axon degeneration

Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy

Reduction of Nuak1 Decreases Tau and Reverses Phenotypes in a Tauopathy Mouse Model

Targeted suppression of mTORC2 reduces seizures across models of epilepsy

Pumilio1 Haploinsufficiency Leads to SCA1-like Neurodegeneration by Increasing Wild-Type Ataxin1 Levels

Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1

Targeting nonsense-mediated RNA decay does not increase progranulin levels in the Grn R493X mouse model of frontotemporal dementia

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

RAS–MAPK–MSK1 pathway modulates ataxin 1 protein levels and toxicity in SCA1

Differential effects of SARM1 inhibition in traumatic glaucoma and EAE optic neuropathies

PFTK1 kinase regulates axogenesis during development via RhoA activation

NMNAT2 supports vesicular glycolysis via NAD homeostasis to fuel fast axonal transport

Reduction of matrix metalloproteinase 9 (MMP-9) protects motor neurons from TDP-43-triggered death in rNLS8 mice

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