Sustained therapeutic benefits by transient reduction of TDP-43 using ENA-modified antisense oligonucleotides in ALS/FTD mice

The abnormal aggregation of TDP-43 into cytoplasmic inclusions in affected neurons is a pathological hallmark of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Although how TDP-43 forms cytoplasmic aggregates and causes neurodegeneration in patients with ALS/FTD remains uncle...

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Detalles Bibliográficos
Autores principales: Takeuchi, Toshihide, Maeta, Kazuhiro, Ding, Xin, Oe, Yukako, Takeda, Akiko, Inoue, Mana, Nagano, Seiichi, Fujihara, Tsuyoshi, Matsuda, Seiji, Ishigaki, Shinsuke, Sahashi, Kentaro, Minakawa, Eiko N., Mochizuki, Hideki, Neya, Masahiro, Sobue, Gen, Nagai, Yoshitaka
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9925842/
https://www.ncbi.nlm.nih.gov/pubmed/36817728
http://dx.doi.org/10.1016/j.omtn.2023.01.006

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