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

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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Bibliographic Details
Main Authors: Toshihide Takeuchi, Kazuhiro Maeta, Xin Ding, Yukako Oe, Akiko Takeda, Mana Inoue, Seiichi Nagano, Tsuyoshi Fujihara, Seiji Matsuda, Shinsuke Ishigaki, Kentaro Sahashi, Eiko N. Minakawa, Hideki Mochizuki, Masahiro Neya, Gen Sobue, Yoshitaka Nagai
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
TDP-43
antisense oligonucleotides
amyotrophic lateral sclerosis
frontotemporal dementia
therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123000094

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http://www.sciencedirect.com/science/article/pii/S2162253123000094

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