Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse model

• Автори: Chunling Duan (Автор), Moorim Kang (Автор), Xiaojie Pan (Автор), Zubao Gan (Автор), Vera Huang (Автор), Guanlin Li (Автор), Robert F. Place (Автор), Long-Cheng Li (Автор)
• Формат: Книга
• Опубліковано: Elsevier, 2024-03-01T00:00:00Z.
• Предмети: article
• Онлайн доступ: Connect to this object online.

Antisense oligonucleotides (ASOs) were the first modality to pioneer targeted gene knockdown in the treatment of amyotrophic lateral sclerosis (ALS) caused by mutant superoxide dismutase 1 (SOD1). RNA interference (RNAi) is another mechanism of gene silencing in which short interfering RNAs (siRNAs)...