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Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9

Cell, 2017-08, Vol.170 (5), p.899-912.e10

2017

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Loss of MBNL Leads to Disruption of Developmentally Regulated Alternative Polyadenylation in RNA-Mediated Disease

Molecular cell, 2014-10, Vol.56 (2), p.311-322

2014

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Transcriptome–pathology correlation identifies interplay between TDP-43 and the expression of its kinase CK1E in sporadic ALS

Acta neuropathologica, 2018-09, Vol.136 (3), p.405-423

2018

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Distinct brain transcriptome profiles in C9orf72-associated and sporadic ALS

Nature neuroscience, 2015-08, Vol.18 (8), p.1175-1182

2015

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Mouse Models of C9orf72 Hexanucleotide Repeat Expansion in Amyotrophic Lateral Sclerosis/ Frontotemporal Dementia

Frontiers in cellular neuroscience, 2017-07, Vol.11, p.196-196

2017

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Muscleblind-like 2-Mediated Alternative Splicing in the Developing Brain and Dysregulation in Myotonic Dystrophy

Neuron (Cambridge, Mass.), 2012-08, Vol.75 (3), p.437-450

2012

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Distinct and shared functions of ALS-associated proteins TDP-43, FUS and TAF15 revealed by multisystem analyses

Nature communications, 2016-07, Vol.7 (1), p.12143-12143, Article 12143

2016

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Protein-RNA Networks Regulated by Normal and ALS-Associated Mutant HNRNPA2B1 in the Nervous System

Neuron (Cambridge, Mass.), 2016-11, Vol.92 (4), p.780-795

2016

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HNRNPA1-induced spliceopathy in a transgenic mouse model of myotonic dystrophy

Proceedings of the National Academy of Sciences - PNAS, 2020-03, Vol.117 (10), p.5472-5477

2020

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MBNL Sequestration by Toxic RNAs and RNA Misprocessing in the Myotonic Dystrophy Brain

Cell reports (Cambridge), 2015-08, Vol.12 (7), p.1159-1168

2015

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Transcriptional profiling of HERV-K(HML-2) in amyotrophic lateral sclerosis and potential implications for expression of HML-2 proteins

Molecular neurodegeneration, 2018-08, Vol.13 (1), p.39-25, Article 39

2018

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Progressive impairment of muscle regeneration in muscleblind-like 3 isoform knockout mice

Human molecular genetics, 2013-09, Vol.22 (17), p.3547-3558

2013

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The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1

Nature biomedical engineering, 2021-02, Vol.5 (2), p.157

2021

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CRISPR-Based Gene Editing Techniques in Pediatric Neurological Disorders

Pediatric neurology, 2024-04, Vol.153, p.166-174

2024

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Properties of LINE-1 proteins and repeat element expression in the context of amyotrophic lateral sclerosis

Mobile DNA, 2018-12, Vol.9 (1), p.35-30, Article 35

2018

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Coding of Sound Envelopes by Inhibitory Rebound in Neurons of the Superior Olivary Complex in the Unanesthetized Rabbit

The Journal of neuroscience, 1999-03, Vol.19 (6), p.2273-2287

1999

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Sensitivity to Interaural Temporal Disparities of Low- and High-Frequency Neurons in the Superior Olivary Complex. I. Heterogeneity of Responses

Journal of neurophysiology, 1997-09, Vol.78 (3), p.1222-1236

1997

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RNA-binding proteins in neurodegeneration: Seq and you shall receive

Trends in neurosciences (Regular ed.), 2015-04, Vol.38 (4), p.226-236

2015

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Sources of the scalp-recorded amplitude-modulation following response

Journal of the American Academy of Audiology, 2002-04, Vol.13 (4), p.188

2002

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Responses of Neurons in the Ventral Nucleus of the Lateral Lemniscus to Sinusoidally Amplitude Modulated Tones

Journal of neurophysiology, 2006-11, Vol.96 (5), p.2388-2398

Batra, Ranjan

2006

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Neural Sensitivity to Interaural Time Differences: Beyond the Jeffress Model

The Journal of neuroscience, 2000-02, Vol.20 (4), p.1605-1615

2000

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Efficiency in Brain Removal Techniques: A Potentially Novel Approach

The FASEB journal, 2020-04, Vol.34 (S1), p.1-1

2020

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Sensitivity to Interaural Temporal Disparities of Low- and High-Frequency Neurons in the Superior Olivary Complex. II. Coincidence Detection

Journal of neurophysiology, 1997-09, Vol.78 (3), p.1237-1247

1997

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Global insights into alternative polyadenylation regulation

RNA biology, 2015-06, Vol.12 (6), p.597-602

2015

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Partners in crime: bidirectional transcription in unstable microsatellite disease

Human molecular genetics, 2010-04, Vol.19 (R1), p.R77-R82

2010

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Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9

Loss of MBNL Leads to Disruption of Developmentally Regulated Alternative Polyadenylation in RNA-Mediated Disease

Transcriptome–pathology correlation identifies interplay between TDP-43 and the expression of its kinase CK1E in sporadic ALS

Distinct brain transcriptome profiles in C9orf72-associated and sporadic ALS

Mouse Models of C9orf72 Hexanucleotide Repeat Expansion in Amyotrophic Lateral Sclerosis/ Frontotemporal Dementia

Muscleblind-like 2-Mediated Alternative Splicing in the Developing Brain and Dysregulation in Myotonic Dystrophy

Distinct and shared functions of ALS-associated proteins TDP-43, FUS and TAF15 revealed by multisystem analyses

Protein-RNA Networks Regulated by Normal and ALS-Associated Mutant HNRNPA2B1 in the Nervous System

HNRNPA1-induced spliceopathy in a transgenic mouse model of myotonic dystrophy

MBNL Sequestration by Toxic RNAs and RNA Misprocessing in the Myotonic Dystrophy Brain

Transcriptional profiling of HERV-K(HML-2) in amyotrophic lateral sclerosis and potential implications for expression of HML-2 proteins

Progressive impairment of muscle regeneration in muscleblind-like 3 isoform knockout mice

The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1

CRISPR-Based Gene Editing Techniques in Pediatric Neurological Disorders

Properties of LINE-1 proteins and repeat element expression in the context of amyotrophic lateral sclerosis

Coding of Sound Envelopes by Inhibitory Rebound in Neurons of the Superior Olivary Complex in the Unanesthetized Rabbit

Sensitivity to Interaural Temporal Disparities of Low- and High-Frequency Neurons in the Superior Olivary Complex. I. Heterogeneity of Responses

RNA-binding proteins in neurodegeneration: Seq and you shall receive

Sources of the scalp-recorded amplitude-modulation following response

Responses of Neurons in the Ventral Nucleus of the Lateral Lemniscus to Sinusoidally Amplitude Modulated Tones

Neural Sensitivity to Interaural Time Differences: Beyond the Jeffress Model

Efficiency in Brain Removal Techniques: A Potentially Novel Approach

Sensitivity to Interaural Temporal Disparities of Low- and High-Frequency Neurons in the Superior Olivary Complex. II. Coincidence Detection

Global insights into alternative polyadenylation regulation

Partners in crime: bidirectional transcription in unstable microsatellite disease

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