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

• المؤلفون الرئيسيون: Chunling Duan (مؤلف), Moorim Kang (مؤلف), Xiaojie Pan (مؤلف), Zubao Gan (مؤلف), Vera Huang (مؤلف), Guanlin Li (مؤلف), Robert F. Place (مؤلف), Long-Cheng Li (مؤلف)
• التنسيق: كتاب
• منشور في: Elsevier, 2024-03-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: 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)...