G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons-Reference-Cited by-同舟云学术

G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons

Published:2023-11-29 Issue:1 Volume:147 Page:
ISSN:0001-6322
Container-title:Acta Neuropathologica
language:en
Short-container-title:Acta Neuropathol

Author:

Rothstein Jeffrey D.,Baskerville Victoria,Rapuri Sampath,Mehlhop Emma,Jafar-Nejad Paymaan,Rigo Frank,Bennett Frank,Mizielinska Sarah,Isaacs Adrian,Coyne Alyssa N.^ORCID

Funder

Robert Packard Center for ALS Research, Johns Hopkins University

NIH NINDS/NIA

Target ALS

Publisher

Springer Science and Business Media LLC

Subject

Cellular and Molecular Neuroscience,Neurology (clinical),Pathology and Forensic Medicine

Link

https://link.springer.com/content/pdf/10.1007/s00401-023-02652-3.pdf

Reference49 articles.

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2. Baskerville V, Rapuri S, Mehlhop E, Coyne AN (2023) SUN1 facilitates CHMP7 nuclear influx and injury cascades in sporadic amyotrophic lateral sclerosis. Brain. https://doi.org/10.1093/brain/awad291

3. Baxi EG, Thompson T, Li J, Kaye JA, Lim RG, Wu J et al (2022) Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines. Nat Neurosci 25:226–237. https://doi.org/10.1038/s41593-021-01006-0

4. Brown AL, Wilkins OG, Keuss MJ, Hill SE, Zanovello M, Lee WC et al (2022) TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A. Nature 603:131–137. https://doi.org/10.1038/s41586-022-04436-3

5. Chew J, Cook C, Gendron TF, Jansen-West K, Del Rosso G, Daughrity LM et al (2019) Aberrant deposition of stress granule-resident proteins linked to C9orf72-associated TDP-43 proteinopathy. Mol Neurodegener 14:9. https://doi.org/10.1186/s13024-019-0310-z

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