Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion
Open accessAbstract
Antisense oligonucleotides can correct disease-specific phenotypes in cultured motor neurons differentiated from iPSCs derived from ALS patients with a C9ORF72 repeat expansion.
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The GGGGCC repeat expansion suppresses the production of C9ORF72 protein by inhibiting transcription.
“The GGGGCC repeat expansion suppresses the production of C9ORF72 protein by inhibiting transcription (140, 153, 225, 369, 497, 520).”
From Blood-Brain Barrier: From Physiology to Disease and Back (2019) · cites this paper as [520]
The Results report that C9ORF72 expression was not significantly decreased in patient cells and that transcription shifted toward the repeat-containing allele. The Discussion likewise says the mutant allele was preferentially transcribed rather than suppressed in patient-derived motor neurons. The paper does not establish reduced C9ORF72 protein production, and its transcription findings run contrary to the claimed mechanism. Page numbers are unavailable in our copy. (LLM, conf=0.88)
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References 51 · Cited by 670
- Authors
- Sareen, D., O’Rourke, J. G., Meera, P., Muhammad, A. K. M. G., Grant, S., Simpkinson, M., Bell, S., Carmona, S., Ornelas, L., Sahabian, A., Gendron, T., Petrucelli, L., Baughn, M., Ravits, J., Harms, M. B., Rigo, F., Bennett, C. F., Otis, T. S., Svendsen, C. N., & Baloh, R. H.
- Affiliations
- Cedars-Sinai Medical Center (US)
- University of California, Los Angeles (US)
- Mayo Clinic in Florida (US)
- University of California San Diego (US)
- Washington University in St. Louis (US)
- Ionis Pharmaceuticals (United States) (US)
- Corresponding author
- Baloh, Robert H.
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2013-10-23
- Volume
- 5
- Issue
- 208
- Pages
- 208ra149-208ra149
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC4090945
- OpenAlex
- W2063996210
- Cited by
- 670
- References
- 51
- Citations by year
- 2026: 19 · 2025: 21 · 2024: 42 · 2023: 28 · 2022: 35 · 2021: 69 · 2020: 64 · 2019: 62 · 2018: 61 · 2017: 58 · 2016: 65 · 2015: 79 · 2014: 61 · 2013: 6
- FWCI
- 44.67
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Funders
- National Institute on Aging (R01AG026251)
- National Institute of Neurological Disorders and Stroke (R01NS063964, R21NS074121, R01NS069669, RC2NS069422, R01NS077402, NS055980, K08NS075094, NS069669, R21NS084528, U24NS07837, K08NS055980)
- National Institute of Environmental Health Sciences (R01ES020395, R01 ES20395)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (P30AR057230)
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Hereditary Neurological Disorders
- Keywords
- amyotrophic lateral sclerosis, C9orf72 repeat expansion, RNA foci, iPSC-derived motor neurons, hnRNP A1, Purα, RNA metabolism, DPP6, membrane excitability, antisense oligonucleotides, gain-of-function toxicity, familial ALS
- MeSH terms
- C9orf72 Protein, Amyotrophic Lateral Sclerosis, Exons, Humans, Motor Neurons, Proteins, RNA, Transcription, Genetic, Oligonucleotides, Antisense, DNA Repeat Expansion, Gene Knockdown Techniques, Induced Pluripotent Stem Cells
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