Author

Emma Mehlhop

Johns Hopkins University

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Neuroscience· 1

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Approach:experimental assay· 1 Organism:human· 1

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Adrian M. IsaacsiD· 1 shared Alyssa N. CoyneiD· 1 shared Frank Bennett· 1 shared Frank RigoiD· 1 shared Jeffrey D. RothsteiniD· 1 shared Paymaan Jafar‐NejadiD· 1 shared Sampath RapuriiD· 1 shared Sarah MizielinskaiD· 1 shared

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2023-2023 · peak 1/yr

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1 shared paperNeuroscience

Adrian M. IsaacsORCID

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1 paper

Recent·Most cited

NeurosciencebioRxivPreprintJun 26, 2023

G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in C9orf72 ALS/FTD human neurons

Jeffrey D. Rothstein, Victoria Baskerville, Sampath Rapuri…·Alyssa N. Coyne

This study shows that targeting G2C4 antisense repeat RNA, not G4C2 sense RNA, mitigates TDP-43 dysfunction in C9orf72 ALS/FTD patient neurons, explaining the failure of previous G4C2 ASO clinical trials.

DOI 10.1101/2023.06.26.546581

humanexperimental assay

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