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A cyclic pyrrole-imidazole polyamide reduces pathogenic RNA in CAG/CTG triplet repeat neurological disease models

Expansion of CAG and CTG (CWG) triplet repeats causes several inherited neurological diseases. The CWG repeat diseases are thought to involve complex pathogenic mechanisms through expanded CWG repeat–derived RNAs in a noncoding region and polypeptides in a coding region, respectively. However, an ef...

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Detalles Bibliográficos
Autores principales: Ikenoshita, Susumu, Matsuo, Kazuya, Yabuki, Yasushi, Kawakubo, Kosuke, Asamitsu, Sefan, Hori, Karin, Usuki, Shingo, Hirose, Yuki, Bando, Toshikazu, Araki, Kimi, Ueda, Mitsuharu, Sugiyama, Hiroshi, Shioda, Norifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10645379/
https://www.ncbi.nlm.nih.gov/pubmed/37707954
http://dx.doi.org/10.1172/JCI164792