CLIP-Seq analysis enables the design of protective ribosomal RNA bait oligonucleotides against C9ORF72 ALS/FTD poly-GR pathophysiology

Amyotrophic lateral sclerosis and frontotemporal dementia patients with a hexanucleotide repeat expansion in C9ORF72 (C9-HRE) accumulate poly-GR and poly-PR aggregates. The pathogenicity of these arginine-rich dipeptide repeats (R-DPRs) is thought to be driven by their propensity to bind low-complex...

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Detalles Bibliográficos
Autores principales: Ortega, Juan A., Sasselli, Ivan R., Boccitto, Marco, Fleming, Andrew C., Fortuna, Tyler R., Li, Yichen, Sato, Kohei, Clemons, Tristan D., Mckenna, Elizabeth D., Nguyen, Thao P., Anderson, Eric N., Asin, Jesus, Ichida, Justin K., Pandey, Udai B., Wolin, Sandra L., Stupp, Samuel I., Kiskinis, Evangelos
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2023
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10637751/
https://www.ncbi.nlm.nih.gov/pubmed/37948524
http://dx.doi.org/10.1126/sciadv.adf7997

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10637751/
https://www.ncbi.nlm.nih.gov/pubmed/37948524
http://dx.doi.org/10.1126/sciadv.adf7997

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