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Antisense Proline-Arginine RAN Dipeptides Linked to C9ORF72-ALS/FTD Form Toxic Nuclear Aggregates that Initiate In Vitro and In Vivo Neuronal Death

Neuron (Cambridge, Mass.), 2014-12, Vol.84 (6), p.1213-1225

2014

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Conversion of Mouse and Human Fibroblasts into Functional Spinal Motor Neurons

Cell stem cell, 2011-09, Vol.9 (3), p.205-218

2011

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CRISPR-Cas9 screens in human cells and primary neurons identify modifiers of C9ORF72 dipeptide-repeat-protein toxicity

Nature genetics, 2018-04, Vol.50 (4), p.603-612

2018

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Probing disorders of the nervous system using reprogramming approaches

The EMBO journal, 2015-06, Vol.34 (11), p.1456-1477

2015

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New approaches for direct conversion of patient fibroblasts into neural cells

Brain research, 2017-02, Vol.1656, p.2-13

2017

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ELAVL4, splicing, and glutamatergic dysfunction precede neuron loss in MAPT mutation cerebral organoids

Cell, 2021-08, Vol.184 (17), p.4547-4563.e17

2021

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Directly Reprogrammed Neurons Still Have Some Growing Up to Do

Cell stem cell, 2019-07, Vol.25 (1), p.1-2

2019

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C9ORF72 protein function and immune dysregulation in amyotrophic lateral sclerosis

Neuroscience letters, 2019-11, Vol.713, p.134523-134523, Article 134523

2019

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Repairing the blood-brain barrier

Science (American Association for the Advancement of Science), 2022-02, Vol.375 (6582), p.715-716

2022

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Downregulation of Hsp90 and the antimicrobial peptide Mtk suppresses poly(GR)-induced neurotoxicity in C9ORF72-ALS/FTD

Neuron (Cambridge, Mass.), 2023-05, Vol.111 (9), p.1381-1390.e6

2023

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Central role for PICALM in amyloid-β blood-brain barrier transcytosis and clearance

Nature neuroscience, 2015-07, Vol.18 (7), p.978-987

2015

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RPS25 is required for efficient RAN translation of C9orf72 and other neurodegenerative disease-associated nucleotide repeats

Nature neuroscience, 2019-09, Vol.22 (9), p.1383-1388

2019

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Small Molecules Take a Big Step by Converting Fibroblasts into Neurons

Cell stem cell, 2015-08, Vol.17 (2), p.127-129

2015

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Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS/FTD human induced motor neurons

Nature medicine, 2018-03, Vol.24 (3), p.313-325

2018

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Organoids Develop Motor Skills: 3D Human Neuromuscular Junctions

Cell stem cell, 2020-02, Vol.26 (2), p.131-133

2020

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A CRISPRi/a platform in human iPSC-derived microglia uncovers regulators of disease states

Nature neuroscience, 2022-09, Vol.25 (9), p.1149-1162

2022

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ADAR2 mislocalization and widespread RNA editing aberrations in C9orf72-mediated ALS/FTD

Acta neuropathologica, 2019-07, Vol.138 (1), p.49-65

2019

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A Small-Molecule Inhibitor of Tgf-β Signaling Replaces Sox2 in Reprogramming by Inducing Nanog

Cell stem cell, 2009-11, Vol.5 (5), p.491-503

2009

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A "multi-omics" analysis of blood-brain barrier and synaptic dysfunction in APOE4 mice

The Journal of experimental medicine, 2022-11, Vol.219 (11)

2022

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Modeling pain in vitro using nociceptor neurons reprogrammed from fibroblasts

Nature neuroscience, 2015-01, Vol.18 (1), p.17-24

2015

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Mitigating Antagonism between Transcription and Proliferation Allows Near-Deterministic Cellular Reprogramming

Cell stem cell, 2019-10, Vol.25 (4), p.486-500.e9

2019

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PIKFYVE inhibition mitigates disease in models of diverse forms of ALS

Cell, 2023-02, Vol.186 (4), p.786-802.e28

2023

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The C9ORF72 repeat expansion alters neurodevelopment

Cell reports (Cambridge), 2023-08, Vol.42 (8), p.112983-112983, Article 112983

2023

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Iroquois Proteins Promote Skeletal Joint Formation by Maintaining Chondrocytes in an Immature State

Developmental cell, 2015-11, Vol.35 (3), p.358-365

2015

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Antisense Proline-Arginine RAN Dipeptides Linked to C9ORF72-ALS/FTD Form Toxic Nuclear Aggregates that Initiate In Vitro and In Vivo Neuronal Death

Conversion of Mouse and Human Fibroblasts into Functional Spinal Motor Neurons

CRISPR-Cas9 screens in human cells and primary neurons identify modifiers of C9ORF72 dipeptide-repeat-protein toxicity

Probing disorders of the nervous system using reprogramming approaches

New approaches for direct conversion of patient fibroblasts into neural cells

ELAVL4, splicing, and glutamatergic dysfunction precede neuron loss in MAPT mutation cerebral organoids

Directly Reprogrammed Neurons Still Have Some Growing Up to Do

C9ORF72 protein function and immune dysregulation in amyotrophic lateral sclerosis

Repairing the blood-brain barrier

Downregulation of Hsp90 and the antimicrobial peptide Mtk suppresses poly(GR)-induced neurotoxicity in C9ORF72-ALS/FTD

Central role for PICALM in amyloid-β blood-brain barrier transcytosis and clearance

RPS25 is required for efficient RAN translation of C9orf72 and other neurodegenerative disease-associated nucleotide repeats

Small Molecules Take a Big Step by Converting Fibroblasts into Neurons

p53 is a central regulator driving neurodegeneration caused by C9orf72 poly(PR)

Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS/FTD human induced motor neurons

Organoids Develop Motor Skills: 3D Human Neuromuscular Junctions

A CRISPRi/a platform in human iPSC-derived microglia uncovers regulators of disease states

ADAR2 mislocalization and widespread RNA editing aberrations in C9orf72-mediated ALS/FTD

A Small-Molecule Inhibitor of Tgf-β Signaling Replaces Sox2 in Reprogramming by Inducing Nanog

A "multi-omics" analysis of blood-brain barrier and synaptic dysfunction in APOE4 mice

Modeling pain in vitro using nociceptor neurons reprogrammed from fibroblasts

Mitigating Antagonism between Transcription and Proliferation Allows Near-Deterministic Cellular Reprogramming

PIKFYVE inhibition mitigates disease in models of diverse forms of ALS

The C9ORF72 repeat expansion alters neurodevelopment

Iroquois Proteins Promote Skeletal Joint Formation by Maintaining Chondrocytes in an Immature State

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