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

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

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)...

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Main Authors: Chunling Duan (Author), Moorim Kang (Author), Xiaojie Pan (Author), Zubao Gan (Author), Vera Huang (Author), Guanlin Li (Author), Robert F. Place (Author), Long-Cheng Li (Author)
Format: Book
Published: Elsevier, 2024-03-01T00:00:00Z.
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