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

• Main Authors: Toshihide Takeuchi (Author), Kazuhiro Maeta (Author), Xin Ding (Author), Yukako Oe (Author), Akiko Takeda (Author), Mana Inoue (Author), Seiichi Nagano (Author), Tsuyoshi Fujihara (Author), Seiji Matsuda (Author), Shinsuke Ishigaki (Author), Kentaro Sahashi (Author), Eiko N. Minakawa (Author), Hideki Mochizuki (Author), Masahiro Neya (Author), Gen Sobue (Author), Yoshitaka Nagai (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...